Tracking Off-Label Cancer Treatment Could Ease Future Trials, Remedies

September 6, 2023 08:47:26

French researchers have discovered that tracking the use of off-label and compassionate cancer treatments could aid in the development and future use of anticancer therapies. The scientists collected data from qualifying patients aged 25 years old and younger who received cancer treatment at French pediatric cancer centers from March 2020 to June 2022. Analysis of this data allowed the research team to spot severe adverse drug reactions (ADRs) and determine how minors plus young adults responded to cancer treatments.

Scientists across the globe are constantly working to develop new and innovative cancer therapies for minors as well as young adults, which often become accessible to cancer patients via compassionate use programs or marketing authorization. However, since there is barely any centralized clinical data on the effect of these off–label prescriptions, a French research team set out to collect data on how minors as well as young adults react to these anti-cancer therapies.

All the patients involved in the study had received diagnoses for malignant neoplasms or similar conditions and had taken off-label or compassionate use cancer therapies. The researchers noted that 351 patients were prescribed 55 different compassionate use or off-label novel drugs as part of the Securing Access to Innovative Therapies for Children, Adolescents, and Young Adults with Cancer Used Outside Clinical Trials (SACHA) study in France; 75% of the drugs were single agents while 65% of the drugs were administered on a molecular interaction basis. Some of the most common therapies prescribed to the patients were dabrafenib plus trametinib, trametinib, regorafenib, dordaviprone, crizotinib, larotrectinib, venetoclax, pazopanib, and cabozantinib.

The study indicated that 34% of the study participants reported experiencing grade 2 to grade 4 ADRs and 43% of the side effects experienced by these patients were due to single-agent trametinib. Additionally, 17 patients reported 24 serious adverse drug reactions related to 12 anticancer therapies; six patients experienced delays in treatment due to adverse drug reactions, three patients had to temporarily discontinue their treatment, and six patients were forced to stop treatment permanently.

Of the 230 patients who had brain tumors, lymphomas or solid brain tumors, 25% (57 participants) responded positively to cancer treatment (10 complete responses and 47 partial responses). The study also reported that 54 of the 57 positive anticancer responses were associated with drugs that already had approval from either the U.S. Food and Drug Administration (FDA) or the European Medicines Agency (EMA). However, the other three responses were due to undocumented antitumor activity, the researchers said.

The researchers published their study findings in the “JAMA Network Open” journal.

The findings in this research could be useful to companies such as Renovaro BioSciences Inc. (NASDAQ: RENB), which are focused on developing immunotherapies targeting solid tumors since this information can provide valuable insights into potential drug targets or mechanisms of action.

NOTE TO INVESTORS: The latest news and updates relating to Renovaro BioSciences Inc. (NASDAQ: RENB) are available in the company’s newsroom at

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