A Window of Hope

 Today’s post was written by Bert E. Thomas IV, PhD, MBA, CEO of the Sarcoma Foundation of America.

SFA CEO Bert Thomas, PhD, MBA
SFA CEO Bert Thomas IV, PhD, MBA


There was a long period of time where there seemed to be little to no progress in finding ways to battle the forgotten cancer known as sarcoma. This period of time lasted for decades, though it probably felt more like centuries to the patients and families that were desperately looking for treatment options that never seemed to come. And while sarcoma still takes too many of our loved ones from us, progress in our fight against this terrible disease is finally being made.

Over the last four years, we have seen four new therapies approved for some or all of the Soft Tissue Sarcoma (STS) patient community. Prior to this “window of hope” it had been more than 30 years since any type of drug had been approved for the patients in our community.

A Timeline of Progress

In 2012, Votrient was approved as a second line treatment for STS, providing a new therapeutic option. Votrient is a multi-tyrosine kinase inhibitor that has helped sarcoma patients as well as patients with other cancers.

In 2015, Yondelis was finally approved in the United States after having been available in more than 50 other countries around the world for many years. Yondelis was approved for patients with leiomyosarcoma and liposarcoma, giving doctors a new weapon to use with patients that have these specific sarcoma subtypes.

Early in 2016, Halaven was approved for patients with liposarcoma, again giving doctors another new weapon for patients with liposarcoma. The biggest news of all came late in 2016 when Lartruvo was approved as a first-line treatment in combination with doxorubicin for all STS patients. Lartruvo is an antibody that targets the PDGF1-alpha receptor and represents the first targeted therapy specifically designed and approved for sarcoma patients.


The age of immunotherapy has come to our patients and this is a great development. The SFA is proud to be a funder of the SARC028 trial, the first clinical trial to explore a novel PD-1 immunotherapy, Keytruda, as a single agent in eight different sarcoma sub-types. The results were very promising. In undifferentiated pleomorphic sarcoma and dedifferentiated liposarcoma patients, partial response rates of 44% and 22% were observed. This is comparable or better than results seen in other cancers. The results of this trial have inspired many new clinical trials exploring PD-1 inhibitors in combination with other agents in sarcoma patients to see if combination therapy provides better response rates across more sarcoma subtypes. Work in the PD1 immunotherapy space continues as it was recently announced that a partnership between Lilly (developer of Lartruvo) and Merck (developer of Keytruda) is being expanded to explore combining their agents in a sarcoma patients.

But wait, there is still more good news.

Drugs in Development

If you go to clinicaltrials.gov and search for sarcoma clinical trials that have industry sponsors, you will find 123 open clinical trials. 123! That is an amazing number for a rare disease that for more than 30 years had been an afterthought to pharmaceutical drug development. Experimental therapies are being investigated as new options for sarcoma patients. We may not always get the publicity of other cancers, but the wheels of progress are turning for sarcoma patients. If just 5 percent of those trials result in a new therapy being approved, we could see six new sarcoma treatments over the next decade. That would more than double the new therapeutic options available. That is hope — that is progress.

Keeping the Window of Hope Open

Hope comes in many forms. But for patients fighting an insidious disease like sarcoma, hope genuinely comes in the form of new therapeutic options that can give each patient a chance for more quality years with their loved ones and for some, to be cured of the disease. The more options our doctors have, the more likely it is that patients will see more favorable outcomes. When you stop and think about the new therapies that have been approved and the new drugs that are being developed, it becomes clear that there has never been a time of greater hope for sarcoma patients.

It is important to remember that we cannot rest on these advancements. While we embrace the optimism that comes with progress, we must do everything possible to keep this window of hope open for all patients who are still waiting for their chance at a cure. That is why the SFA continues to fund basic sarcoma research that could evolve into new targets, new experimental therapies, new clinical trials, and, most importantly, new approved therapies to treat sarcoma.