April 6, 2013

MMRF Newsflash from IMW 2013 - Volume 2

This morning, Sagar Lonial, MD, summed up the purpose of the IMW in comments preceding his presentation on Pomalyst (pomalidomide) today: “…what we do here is all about improving outcomes for patients.” This sentiment has been palpable throughout the first couple of days as clinical and translational researchers from around the world have come together to discuss the most pressing research questions related to multiple myeloma.

Over the last few years, an emerging area of focus is the treatment of smoldering multiple myeloma patients. The largest study to date from Mateos et al in Spain has shown a survival benefit for patients treated with Revlimid and dexamethasone, but while promising, more data from additional trials are needed to understand the overall risk-benefit ratio of treating patients earlier, and, most importantly, and there was a certain amount of consensus here, how can we prospectively identify high-risk smoldering patients – those most likely to convert to active myeloma within two years – as opposed to lower risk patients who may not convert for up to 10 years.

There have also been updates on the newest therapies – Pomalyst and Kyprolis (carfilzomib). For Pomalyst, a sub-analysis of the pivotal Phase II (which was conducted in partnership with MMRC) showed that patients with deletion 17p (chromosomal abnormality), a group of patients who are not as likely to respond to standard therapies, can benefit from treatment with this drug. Further, Pomalyst provides end-organ function benefit. For Kyprolis, Andrzej Jakubowiak presented a sub-analysis from the MMRC Kyprolis-Rev-dex trial in newly diagnosed patients that showed that elderly patients were just as likely to obtain high response rates (85% NCR and CR) though they were also more likely to need dose reductions. There was also very preliminary work presented looking on the combination in high-risk SMM patients.

Finally, the question of maintenance therapy continues to be an important one, and as more data emerge, it is clear that there is a consistent decrease in risk of disease progression, though there is a slightly increased risk of secondary cancers (primarily MDS/AML). However, the risk of disease progression is much higher. Building on a theme mentioned in yesterday’s entry, we really need to identify which patients are likely to have the greatest benefit with the lowest risk. Data show that those patients in a complete response (CR) following high-dose chemo and stem cell transplant do not really benefit from maintenance (as opposed to those who do not initially reach CR). Once again, we are back to really needing to better match patients to treatments that are most likely to benefit them and are associated with the lowest risk of side effects – which is exactly what we are working to accomplish using CoMMpass data combined with data from similar initiatives. We are getting closer!