Histology-Specific Trials and Drug Development Strategy Pave the Path to Progress in Soft Tissue Sarcoma

The implementation of broad program projects, spanning areas of research, drug development, and translational medicine, are needed to better understand the nuances of soft tissue sarcoma, so that care can be personalized to each individual patient, according to William D. Tap, MD.

“The future is really bright. We are entering into yet another era in treatment for patients with sarcoma. We are developing interesting approaches in some of the sarcoma subtypes, and this is how we are going to effect change and improve outcomes,” Tap said. “This is a very rare, heterogenous disease, so getting enough information to understand what is driving a cancer and how that cancer is affecting our patients, and partnering with patients, pharmaceutical companies, and researchers to develop the next level of therapies, remains a challenge.”

In an interview with OncLive^®, Tap, chief of the Sarcoma Medical Oncology Service at Memorial Sloan Kettering Cancer Center, discussed the key challenges faced in the treatment of patients with soft tissue sarcoma and efforts being made to overcome them.

OncLive^®: How do you currently approach the treatment of your patients with soft tissue sarcoma in clinical practice?

Tap: It is a difficult question because soft tissue sarcomas represent [approximately] 80 different diseases; it is almost like asking an oncologist how [they] approach cancer. One of the [most promising] paradigm shifts within sarcomas is that we are beginning to understand the nuances of these rare diseases. In doing so, we can come up with some unique treatment opportunities for our patients.

One of the biggest approaches that we are [incorporating], not only at Memorial Sloan Kettering Cancer Center, but in the international community, is developing broad program projects, across areas of research, drug development, and translational medicine. We can pull from this [research] as we begin to understand the different nuances of sarcomas, to [consider] what the right treatment [approach is] for [each] individual patient.

Over the past few years, have any studies helped to move the needle forward?

One of the most important trials was negative. We [initially saw] some positive data from a phase 2 trial that led to the FDA approval of a drug, so our community rapidly got together and performed a large phase 3 study in sarcoma. Unfortunately, it was a negative study, and that galvanized the community to understand why. [One of the reasons is that we] continue to look at this disease as a single-disease entity. More and more, we are moving into histology-specific clinical trials in sarcomas and drug development strategy. Because of that, we are seeing [many] drugs now make it into regulatory phases, and that is exciting.

Recently, there was a drug approved in epithelioid sarcoma, and [we have seen] some wonderful data in a rare disease known as perivascular epithelioid cell neoplasm. We continually see advances made in gastrointestinal stromal tumors, with several drugs approved. We are also seeing more and more of these programs develop internationally, and that is the most exciting change that has been made over the past few years.

Could you expand on efforts that are being made to address unmet needs in the realm of sarcoma?

There is so much unmet need. In rare diseases, [we need to] understand the biology and then [cultivate] relationships with the right partners to develop drugs. Oftentimes, our community must band together to show the data and then gain the interest of pharmaceutical partners to develop drugs in rare diseases. Oftentimes, there are misperceptions about the market and the need, but there are, [in fact], tremendous needs within the patient population. This is one [area] that we are trying to advocate for the most.

[Another area of interest is] community building with patients. Getting a diagnosis of a rare cancer can be daunting and difficult, and there is not a lot of information out there. [As such, this effort is focused on] helping to direct patients to the right treatment centers and the right treatments.

Finally, we are interested in understanding how cancers affect those who are in the adolescent and young adult [age] range. Sarcomas tend to have high proportion of patients who are young, so understanding the unique psychosocial needs that affect these patients could help us to improve our treatments for them.

What would you like to impart to your colleagues?

We have had inroads in understanding how to manage patients with sarcoma, but a tremendous number of nuances remain. For example, [if you consider] liposarcoma, which is a cancer of fat, about 3 or 4 different variations of this disease [exist], and patients require different treatments based on the variation that they have.

We like to partner with our colleagues in the community so that we can [better] care for our patients. [We want to] help them understand the diagnosis, the treatments that are available, the unique psychosocial needs of our patient population, and how we can partner with them to deliver care. This is a tight international community, so we all benefit from work that is being done all over the world.