How decades of blood cancer research is paying off

Cancer survival rates around the world continue to improve and now, with more treatments options, earlier diagnosis and greater awareness, people with many forms of blood cancer are living longer and better with their disease than ever before. Thanks to the latest advances in this branch of medicine, the future is looking even brighter.

Gaining a deeper understanding of biology

Our growing understanding of the biology of blood cancers is transforming the development and delivery of treatments for patients. Since the introduction of the first monoclonal antibody for the treatment of blood cancers 20 years ago, the scientific community together with healthcare companies have worked tirelessly to keep improving the options available to people with blood cancer. Some forms of the disease have even been transformed into chronic illnesses that can be managed with ongoing treatment.

Nancy Valente, VP Global Product Development Haematology/Oncology at Roche

There is still however more to do. It is becoming increasingly clear that most blood cancers are not likely to be cured with a single intervention or management approach. But with complexity also comes opportunity; by closely examining the role of various signalling pathways that cause blood cancers to develop and grow, researchers have been able to use this knowledge to identify potential new targets for treatment. This includes looking at a family of proteins called ‘B-cell lymphoma 2’ which play an important role in regulating cell death, as well as developing new small molecule inhibitors to target specific steps in the newly uncovered signalling pathways.

Antibody-drug conjugates (ADCs) are another emerging class of anticancer treatments that are revolutionising chemotherapy for blood cancers. ADCs have a novel mode of action in which a toxin is attached to a monoclonal antibody to target and destroy cancer cells. Unlike traditional chemotherapy, ACDs are designed to bind only to specific receptors and so they are able to spare healthy cells while effectively destroying cancer cells.

We are also starting to see encouraging results looking at chemotherapy-free treatment options. This is particularly important as a possible new treatment route for the many patients facing relapsed disease, or disease that has not responded to initial treatment (refractory) but who – due to age / frailty or comorbidities – are not well enough to undergo current standard-of-care chemotherapy.

At Roche we are driven by our passion for understanding the biology of disease, and by our desire to do more for patients. What is important is that researchers, health authorities, physicians and industry work together to apply innovative thinking to new technologies and approaches, said Nancy Valente, VP Global Product Development Haematology/Oncology at Roche
Reimagining clinical trial design

It’s not only new treatment options that are advancing the science of haematology. With so many new potential treatment targets emerging, the design of clinical studies is also becoming increasingly important. Trials must ensure that the clinical value of a potential new treatment is determined accurately and efficiently so that new medicines can reach the patients that need them as quickly as possible. This has led to important changes in the definition of, and use of endpoints in clinical trials.

As treatments have become more effective, conventional endpoints in oncology trials, such as median progression-free survival (how long a patient lives without their disease worsening) and overall survival (total time a patient remains alive), can now take a long time to measure, especially in slower growing blood cancers, such as follicular lymphoma. This is where the use of newer ‘surrogate endpoints’ comes in. Surrogate endpoints are measures that predict the clinical benefit of a treatment (like overall survival) but are much quicker to evaluate. One example is the measurement of ‘minimal residual disease (MRD)’ in chronic lymphocytic leukaemia.

Minimal residual disease (MRD)

MRD refers to the small amount of cancer cells that can remain in a patient’s blood or bone marrow after treatment. MRD is a major cause of relapse. The emergence of MRD as an important endpoint in clinical trials is significant as it allows clinicians to assess the extent to which a treatment is working, whether a patient is likely to relapse or if they have achieved deep remission.

The way that treatments are administered is another area where scientific advances stand to improve the lives of patients. People facing treatment for blood cancer often have to contend with treatments that take a long time to receive, such as lengthy intravenous (IV) infusions, and the prospect that treatment could be ongoing for the remainder of their life. The introduction of faster, more convenient administration options can therefore meaningfully improve the patient treatment experience. Subcutaneous administration for example, whereby treatment is delivered via injection under the skin, has the potential to significantly reduce treatment times for certain blood cancers. With comparable efficacy to the more traditional IV, studies show that both patients and healthcare professionals prefer SC administration due to reductions in clinic time and emotional distress, as well as increased comfort and convenience.1,2,3 This is important as it represents a way of helping patients preserve the highest quality of life possible while managing their disease.

What we are seeing at the moment is a real period of dynamism in the science of haematology, bringing fresh hope to people affected by diseases of the blood, said Nancy Valente, VP Global Product Development Haematology/Oncology at Roche
Innovations in drug delivery


  1. Lugtenburg et al. Haematologica. 2015; 100: S483

  2. Rummel M et al. Ann Oncol. 2016; 28(4): 836-842

  3. Sayyed et al., Haematologica 2012; 97(Suppl. 1): Abstract P1749

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