FDA Grants Rare Pediatric Disease Designation to LSTA1 in Osteosarcoma

The Food and Drug Administration (FDA) has granted a Rare Pediatric Disease Designation (RPDD) to the investigational drug LSTA1 for the treatment of osteosarcoma — a rare cancer known to develop in children as well as adolescents and young adults — according to a news release from pharmaceutical company Lisata Therapeutics.

Osteosarcoma, which starts in the bones, accounts for approximately 2% of childhood cancers, according to the American Cancer Society, which stated that there are approximately 1,000 new cases of the disease each year in the United States, about half of which are in children and teens.

“LSTA1 has already demonstrated promise in preclinical and clinical settings for the treatment of several advanced solid tumors, and we believe that potential extends to a beneficial impact in osteosarcoma, a rare cancer that usually develops in the osteoblast cells that form bone,” said Dr. Kristen K. Buck, executive vice president of research and development and chief medical officer of Lisata, in the news release. “Receiving RPDD from the FDA is an important milestone as we plan future clinical expansion for LSTA1, and, more importantly, it underscores the high unmet medical need of this patient population.”

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According to the FDA, under its Rare Pediatric Disease Priority Review Voucher program, the agency determines if treatments meet the definition for treating rare pediatric diseases in order to grant a designation which “may provide the opportunity for products to qualify for a priority review voucher.”

LSTA1, according to Lisata, “is an investigational drug designed to activate a novel uptake pathway that allows co-administered or tethered (i.e., covalently bound) anti-cancer drugs to penetrate solid tumors more effectively.” The drug also has the potential to modify the tumor microenvironment in order to make a tumor more susceptible to treatment via immunotherapy, the company stated.

In addition to the Rare Pediatric Drug Designation, LSTA1 has previously received orphan drug designation for the treatment of pancreatic cancer and glioblastoma multiforme, as well as a Fast Track designation from the agency for patients with pancreatic cancer.

Lisata, according to the press release, has accumulated “significant non-clinical data demonstrating enhanced delivery of a range of existing and emerging anti-cancer therapies, including chemotherapeutics, immunotherapies and RNA-based therapeutics. … Lisata is exploring the potential of LSTA1 to enable a variety of treatment modalities to treat a range of solid tumors more effectively.”

According to the American Cancer Society, among children, teens and young adults with osteosarcoma the disease typically starts in the areas of the body where bone grows quickly, including the ends of the bones in the arms or the legs. Most tumors, they state, develop in the bones around the knee, and the upper arm bone near the shoulder is the second most common tumor site.

The five-year survival rate (the percentage of patients who will still be alive five years after diagnosis) for osteosarcoma is 59%, according to the American Cancer Society, based on patients who received a diagnosis of osteosarcoma between 2012 and 2018.

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