MSK at ASCO: Targeted Therapy Significantly Delays Cancer Progression in Rare, Aggressive Sarcoma

An oral targeted therapy is showing promise for people with dedifferentiated liposarcoma (DDLPS), a rare subtype of soft tissue sarcoma, according to Memorial Sloan Kettering Cancer Center (MSK) physician-scientists. Results from the long awaited SARC041 phase 3 clinical trial were presented today at the annual American Society of Clinical Oncology (ASCO) meeting.

Investigators reported that patients receiving abemaciclib lived a median of 10 months without their cancer progressing, compared with about 1.5 months in the placebo group. The multicenter trial (NCT04967521) enrolled 108 patients across nine centers. Experts say the findings will help set a new standard treatment option for people with recurrent or metastatic DDLPS.

"These findings are especially encouraging because they show a targeted therapy can meaningfully delay disease progression in a cancer that has long been challenging to treat," said Mark A. Dickson, MD, an MSK sarcoma medical oncologist, trial investigator, and plenary presenter. The clinical trial was built upon on earlier research that suggested DDLPS may be especially vulnerable to CDK4 inhibition because the CDK4 gene is amplified in most DDLPS tumors, added Dr. Dickson.

Abemaciclib is part of a class of drugs called kinase inhibitors and is currently approved for use in certain breast cancers. The treatment works by blocking CDK4 and CDK6, proteins which cancer cells are fueled by and need to grow and divide. Interfering here means the drug can slow, and in some cases prevent, the tumor from growing and moving into other areas of the body.

DDLPS is most often diagnosed in the abdomen or retroperitoneum (the anatomical area in the back of the abdomen located directly behind the peritoneum) and presents significant treatment challenges once it recurs or spreads. Current treatments have only provided a modest benefit, motivating doctors to seek better therapeutic options for their patients.

In 2021, just as she was preparing for retirement, MSK patient Andi Straus was diagnosed with a rare liposarcoma. After surgery, she faced multiple recurrences, surgeries, chemotherapy, and an increasingly difficult prognosis. A year later, she enrolled in the clinical trial that she credits with giving her four additional years of quality life. Despite minor side effects, Andi is doing well and recently celebrated her 75^th birthday surrounded by family and friends at a brunch in her honor. "When I was first diagnosed, my surgeon told me I had a 50-50 chance of being alive in five years," she shared. "Every day since has felt like an indescribable miracle-one I'm profoundly grateful for."

Data Breakdown:

• Patients receiving abemaciclib had a median progression-free survival of 9.7 months, compared with 1.5 months for placebo.
• Tumors shrank in some patients receiving the drug, while no responses were seen in the placebo group.
• An encouraging trend toward improved overall survival was observed.
• No new safety concerns.

About DDLPS:

• Soft tissue sarcomas are rare cancers that represent less than 1% of all adult cancers. DDLPS is a rare and aggressive subtype of this disease group. ACS
• DDLPS most often occurs in adults between the ages of 50 and 75 and affects men and women at similar rates. Cure Sarcoma
• DDLPS often recurs following surgery and can metastasize, making long-term disease control difficult.
• 5-year survival rates vary widely depending on where the cancer develops and whether it has spread, but advanced disease is associated with poorer outcomes.

The phase 3 study was supported by Eli Lilly and Company; Cycle for Survival; Mitchell Goodman Fund for Sarcoma Research; Peter and Devon Briger Foundation; Heather and Jordan Turkewitz; and the National Cancer Institute. See here for ASCO 2026 LBA2. Dr. Dickson reports no disclosures during this time.

CONTACT:
Courtney Nowak
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347-931-6814