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Haematology: How far we’ve come, and how far we have to go

Haematology: How far we’ve come, and how far we have to go

In a Special Report, Patrick Laroche, Janssen EMEA Haematology Therapy Area Lead, looks at how haematology is leading the way in oncology innovation, how CAR-T has the potential to revolutionise treatment for multiple myeloma, and the challenges that lie ahead in this field.

Over the last decade, treatment for blood cancers has changed significantly, transforming expectations of what a diagnosis means for patients. A new decade is now beginning, and I believe we are sitting at a truly exciting moment of exponential scientific development in cancer specifically.

I liken it to the feeling I had working in HIV in the 1990s, a disease in which we have now made incredible strides, completely transforming its disease trajectory.

This moment has inspired me to really reflect on some of the exciting data and innovative treatments that will be potentially available in clinical practice, and what this could really mean for patients and their families; treatments that have the potential to change lives by helping blood cancer patients improve their outcomes.

As was evident at the American Society of Hematology (ASH) congress in December 2019, we continue to see incredible scientific development in the field.

In some diseases, like multiple myeloma (MM), a remarkable improvement in survival outcomes has been achieved in the last 10 years. We are now able to roughly double the time without progressive disease for newly-diagnosed patients, thanks to the introduction of triplet and quadruplet therapies, and we expect more innovative treatments in clinical practice in the very near future.

Progress not only concerns therapies but also improvements in imaging, diagnosis, and the emergence of potential surrogate markers, such as the ability to obtain clinical minimal residual disease (MRD) measurements from peripheral blood samples instead of requiring bone marrow biopsy. This brings both benefits for patients and the possibility to more easily track some disease markers.

Chimeric antigen receptor T-cell therapy (CAR-T) was the treatment class that really took centre stage at the congress. In my opinion, precision medicines like CAR-T therapy are one of the most exciting advances in blood cancer treatment and represent the future of healthcare for certain diseases. They allow us to abandon the traditional ‘one-size-fits-all’ approach to cancer research and development, and instead truly focus on finding the right treatment for the right patient at the right time.

Traditionally, medicines and biologics are manufactured in large batches for many patients and stored until they are needed. CAR-T is different.

The process for manufacturing CAR-T involves collecting blood from each patient and developing a therapy specific to them within just a few weeks. By harnessing the power of a patient’s own immune system to seek and destroy cancer cells, CAR-T has the potential to revolutionise the way healthcare professionals treat blood cancers, and now we’re starting to see that potential become reality.

At ASH, data from long-term follow-up studies confirmed CAR-T’s clinical importance in both diffuse large B-cell lymphomas (DLBCL) and acute lymphoblastic leukaemia (ALL), and positive new data were presented supporting its expansion as a potential treatment for MM. These therapies will become increasingly important as they provide new options for patients.

However, while the therapeutic promise of CAR-T for blood cancers and beyond is fantastic, companies and health systems face unprecedented procedural challenges to ensure it lives up to its potential.

With CAR-T, hospital sites are both a customer and a supplier, requiring auditing and certification. Each batch is unique to one patient, so traceability and transparency are vital, requiring a robust tracking system to secure the chain of identity and chain of custody. Patient management is complex, particularly for MM patients, so many stakeholders will be involved before, during, and after the treatment. To meet these challenges requires extensive collaboration between healthcare professionals (HCPs), drug manufacturers, regulatory bodies, other development partners and, critically, collaboration with patients and patient advocacy groups (PAGs). Given the unique nature of the treatment, it has been vital to engage with PAGs from a very early stage in the development process to ensure we address patient needs.

If we get it right, there is so much potential, and hope for transforming people’s experience of cancer. At ASH, we saw targeted therapies, monoclonal antibodies and cell therapies are now in some cases even opening up the potential for a cure.

Looking further ahead, I am also excited to see how disease interception strategies will help us to not just treat, but prevent cancer.

Instead of reacting to cancer once people become ill, we are looking at how to treat individuals earlier in their disease – potentially even before the onset of symptoms. This cancer interception approach is working to unlock new ways to detect and stop cancer before it can take hold in the body. This means addressing the root causes of disease; intervening earlier than today’s clinically accepted point of diagnosis; and seeking solutions that stop, reverse or inhibit progression to that specific disease.

Finally, and perhaps most importantly, coming together with all the delegates from around the world at ASH always reminds me of the power of collaboration – especially with the HCPs, advocates and caregivers who are supporting patients day in and day out. This is, I believe, what will help us to harness this moment of rapid scientific development and address one of the most difficult health challenges: defeating cancer for good.

This article originally appeared on Thepharmaletter on January 28 2020.