A Longer Long-Term: The Rise of Survivorship
With most blood cancers, the treatment decisions made upon initial diagnosis may have the most impact. If we develop better medicines and help facilitate informed treatment decisions early on, we can potentially change the course of disease progression.
The American Society of Hematology (ASH) annual meeting is in my own “backyard” of San Francisco this year. ASH always reinforces my hope that many of the medicines being discussed now will have a meaningful impact on people for years to come. The new insights we’re gaining from continuing research could help us speed development of important new medicines that could help improve outcomes for people with blood cancer.
The past century saw many breakthroughs in the treatment of blood cancers. The first targeted cancer therapy ever approved by the FDA was a monoclonal antibody for treating a blood cancer, and data continues to roll in more than 15 years after that approval. Moving forward, hematologists are in a unique position to shape the discussion around long-term outcomes, due in part to advances in blood cancer research that have transformed many of these diseases from fatal conditions into chronic ones.
An unprecedented amount of data (including numerous presentations from ASH meetings) is now available to help us understand how early results relate to outcomes many years later. By providing earlier answers about how effective a medicine is against a given disease, these measures may act as “surrogates” for endpoints that take longer to measure, such as overall survival.
While discussions about long-term outcomes are not new, it's only recently that we have been able to harness this data to develop novel endpoints for early treatment decisions or to accelerate access to new therapies.
While overall survival remains the gold standard in measuring clinical benefit, it can take a very long time to measure, especially for blood cancers that are less aggressive. Other endpoints are like guideposts along the road of blood cancer survivorship. They let us know if we’re on the right track with the decisions we’re making now. Progression free survival, for example, demonstrates that the disease has, at least for a period of time, been stopped in its tracks. Prolonging that time early in the treatment paradigm may have a significant impact on the course of the disease.
The variety of tools available to assess treatment efficacy is expanding substantially. While response rates have long served as indicators of efficacy, we are working in collaboration with health authorities and the hematology community to further refine two more sensitive endpoints that measure response: durable complete responses (CR) and minimal residual disease (MRD). CR is the complete absence of signs and symptoms of the cancer and MRD involves searching for traces of cancer even when standard tests suggest it’s gone.
A durable CR or achievement of MRD negativity may result in completion of treatment early and could predict a longer remission before additional treatment is needed, or even longer survival down the road. For those who are newly diagnosed, the impact on survival may be greatest. Achieving these kinds of responses early in the course of treatment gives us hope that the disease can be halted before it becomes more difficult to treat.
We’re gathering our data and applying what we’ve learned from more than 20 years of research in blood cancers to inform clinical studies of investigational medicines. We are also exploring new combination approaches and sequencing across various treatment settings, through multiple approaches and in partnership with others across the industry.
There is so much more we still need to do, and we work every day to try and bring new and better tools to patients and their doctors. Every time we see new data like that presented at ASH, it enriches our ongoing discussions about these important issues. With many new medicines, we are making solid progress toward helping people live longer with blood cancer, and I look forward to what’s ahead.