My experience at the 64th annual ASH Meeting and Exposition was marked by a series of firsts: My first time attending an industry conference and my first time in New Orleans. In attending various presentations throughout the weekend, I came to understand how the current dynamics surrounding haematology-oncology today will continue to shape the field moving forward:

Gene therapies are game-changers, but require much consideration around their use

Gene therapies with recent FDA approval, such as bluebird bio’s Zynteglo (betibeglogene autotemcel), have significantly shifted current clinical expectations for patients with blood-based diseases like ß-thalassemia, and future expectations for sickle cell disease (SCD), based on their ability to essentially cure patients of their genetic conditions. With this in mind, recent research from Debra Friedman, MD, at Vanderbilt underscores the reality that receiving one of these interventions does not mean a patient is totally in the clear. Her research suggests that patients receiving these treatments may be at a higher risk for developing myeloid malignancies than those who do not.

As a result, even though these interventions are considered curative therapies, clinicians must consider their potential benefits and tradeoffs. For instance, registries to track long-term outcomes could be implemented to bolster existing knowledge around the risk-to-reward ratio of these interventions.

On a positive note, pre-clinical research demonstrated the viability of expanding gene therapies to related indications, such as α-thalassemia. While drug development is a difficult process fraught with pitfalls, the future of gene therapies remains bright.

We’re still smoothing out the bumps of CAR-T administration

Chimeric antigen receptor T-cell (CAR-T) therapies have positively impacted the field of haematology-oncology. In 2022, both Yescarta (axicabtagene ciloleucel) and Breyanzi (lisocabtagene maraleucel) received FDA approvals in 2L diffuse large B-cell lymphoma (DLBCL), an earlier setting than their previous 3L FDA indications. While this is undoubtedly exciting for patients suffering from these diseases, their clinicians, and the caregivers involved in their journeys, many discussions held during ASH touched on the barriers that patients and physicians still face in receiving treatment.

For example, manufacturing limitations, difficulties coordinating care with patients and caregivers, referral pathways to cancer centers, obtaining insurance approvals, and patients’ availability to handle toxicities all represent significant barriers. Many of these are further exacerbated by differences in socioeconomic status, another important consideration to keep in mind when attempting to ensure equitable access to these interventions. In addition, given their recent approval status, it’s important to recognize the importance of gathering real-world evidence to monitor the efficacy and safety of these molecules over time.

There is no doubt that these treatments have further potential to revolutionize the approach to care for patients with blood-based malignancies. Equally as important is considering commercially-viable methods of reducing disparities in access.

Truly understanding the physiological impact of COVID-19 is a long game

Although the pandemic may have receded from many people’s minds, the factors involved in genuinely understanding the long-term health impact of COVID-19 infections are only starting to become clear. In addition to the respiratory system, such infections impact the cardiovascular and circulatory systems, with recent studies intent on determining the best ways to screen for these impacts.

For example, a recent abstract highlighted how biomarkers, including Factor VIII and von Willebrand factor, were elevated in cancer patients who suffered from thromboembolism after COVID-19 infection compared to those who did not. This implies that, for those undergoing treatment for cancer, future screening tools could be implemented to pre-emptively gauge a patient’s risk factors for severe events, potentially improving overall health outcomes.

Moving forward, further research could investigate how and when these screening tools should be implemented at a population level to best determine a specific patient’s risk.

Precision medicine is not just for solid tumors

Recent advances in biomarker testing have ushered in a new era of treatment in oncology. Across indications, pathologists and oncologists are keeping abreast of novel targeted therapies and their associated biomarker mutations. However, it is becoming increasingly evident that these are no longer focused on the world of solid tumors.

For example, research conducted by Mina Xu, MD at Yale demonstrated that utilizing liquid biopsy (LBx)-based next-generation sequencing (NGS) in hematological malignancies, while still nascent, holds future promise. In comparing concordance between tissue-based and LBx assays, her team found about a 74% similarity rate. While this is not yet high enough to be used in clinical settings, the assays detected all tissue mutations, with some differences in subclonal variants. These findings can give clinicians a better, more comprehensive aspect of a tumor’s mutational status compared to testing a single site of tissue.

Furthermore, cutting-edge research conducted at City of Hope demonstrated that whole genome sequencing could determine treatment combinations a patient’s cancer would be most responsive to. In doing so, clinicians were able to decrease the risk of death by 83% in a study population of 35 patients. In addition to corroborating such findings, further studies could establish best practices for optimizing treatment based on a patient’s mutational profile.

Increasing diversity and equity in research and therapeutic access is a priority, but there is still a long way to go

Throughout the conference, many presentations rightfully pointed out the imbalances in how clinical trials recruit and enroll patients, in addition to the contributions that income inequality and geographic spread play in preventing patients from receiving the best care possible. Thankfully, many in the community are working to address these barriers – although the fight is far from over.

Take, for example, diversity initiatives in the FDA. Recent studies on clinical trial enrollment indicate that Black patients are still underrepresented: Although these populations represent over 30% of those with Multiple Myeloma in the US, an investigation of 19 haematology-focused clinical trials demonstrated only 4% of the participants were Black. Increasing representation accordingly is paramount, not only from a health equity perspective but also to better understand differences in disease etiology, which can help boost health outcomes in the long run.

In addition, both Bluebird bio and Vertex Pharmaceuticals are working to apply their gene therapies to SCD. While this is an exciting moment for patients suffering from such a debilitating disease, it also must be pointed out that most of the world’s burden of SCD occurs in sub-Saharan Africa, which is typically late to receive innovative medicines compared to other regions, such as the US or EU5. As such, it is imperative to consider potential pathways for expanding access to high-cost therapies in the global South, ensuring that curative diseases are not limited to countries with significant healthcare spend.

I hope you enjoyed this article! If you have any questions or would like to follow up please feel free to reach out at D.brown@hrwhealthcare.com, or to the wider OR:BIT team at ORBIT@hrwhealthcare.com.

By Dylan Brown



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