Sarcoma Care: Biomarker Advancements Shape the Future

At our recent Institute for Value-Based Medicine® event in Boston, Vinayak Venkataraman, MD, medical oncologist at Dana-Farber Cancer Institute and Harvard Medical School, was a panelist for the discussion, “Recent Advancements in Identifying Predictive Biomarkers for Sarcomas,” that detailed how care and drug development for these rare cancers has expanded over the past 20 years.

Here, he also addresses the impact of value-based care within the sarcoma arena, FDA approvals, and what he sees as the return on investment (ROI) for rare drug development in this space, in which companies often don't want to invest time and the money in developing rare drugs because there may not be an ROI.

Venkatraman’s primary focus is treating patients with bone and soft tissue sarcoma, and he is a board-certified internist and pediatrician, with interest in supporting young adults with cancer.

This transcript has been lightly edited.

Transcript

What are some key takeaways from your presentation, “Recent Advancements in Identifying Predictive Biomarkers for Sarcomas”?

Over the past 2 decades, we've really improved our understanding of the biology and the natural history of sarcomas, and through advancements in next-generation sequencing and other modalities, we've also been able to molecularly characterize the driving force of many sarcomas and also predict whether or not they respond to novel therapies, such as targeted therapies, immune therapies, and cellular therapies. Our talk really [highlighted] using some key examples such as gastrointestinal stromal tumor for which 20-plus years ago imatinib was approved as KIT inhibitor.

Since then we've really advanced our understanding both of primary treatment, as well as why patients develop resistance to imatinib, and then by understanding that biology, creating new medications and new novel therapies that can counteract that resistance. So over the past 2 decades we've really seen an advancement in our approach to managing those patients over time, and so the talk also [highlighted] the need for us to just better understand the biology of our cancers, and part of that is just sending a lot of these testings, such as next-generation sequencing. We don't always know in advance what we're going to find, but oftentimes by sending and collecting more data, we can make advances in the future.

Can you speak to the impact of recent FDA decisions on advancing sarcoma care?

When I think of [return on investment], I think about the patient in front of me and seeing them respond to effective treatment, and we know that our treatments right now are inadequate. They can provide some meaningful progression-free survival, but rarely are they curative or rarely are they durable. So cellular therapy to me, the return on investment is really seeing the patients thrive beyond their cancer, and sort of potentially—we are averse to using the word cure—but some of these treatments have the promise for really durable remissions that could potentially be curative.

I think the first step is just getting these treatments out there and sort of seeing how they do in the real world. We know how they have done in clinical trials, and my hope is that there's a lot of interest in bringing cellular therapies to solid oncology. And so once you have the platform technology and then you can modify it based on the solid tumor subtype, maybe that can improve some of the finances around development of these expensive medications such that even though these are rare diseases, if you have the platform and then you're applying it to specific rare diseases maybe that brings down the overall cost of the treatment.

I can only say, as a doctor, it's just amazing to see these treatments in clinical practice and my hope is that can companies continue to invest in them to benefit our patients.