Researchers find disrupting tumor energy slows cancer growth.
Researchers at the Medical University of South Carolina have discovered a new approach that may change how head and neck cancers are treated. These cancers, which develop in the lining of the mouth, nose, and throat, are often aggressive and resistant to standard treatments. Even when surgery, chemotherapy, or radiation is successful, tumors frequently return, and the treatments can harm healthy tissue, leaving patients with lasting side effects. Scientists at MUSC Hollings Cancer Center have been testing a compound called LCL768, which works in a different way. Instead of broadly attacking cells, it targets the cancer’s energy systems, effectively cutting off the power supply that tumors rely on to survive and grow.
LCL768 is a synthetic version of ceramide, a fat naturally found in cells. Ceramides help maintain cell function and can trigger cell death when a cell is damaged or stressed. Many head and neck tumors have low levels of this fat, which allows them to grow aggressively. The drug increases the amount of a particular type, called C18-ceramide, inside the mitochondria of cancer cells. Mitochondria are the tiny structures that produce energy for cells. When C18-ceramide builds up, it sets off a process called mitophagy, where the cell destroys its own mitochondria. Without mitochondria, cancer cells lose their energy and die.
The compound also interferes with another part of the cell’s energy system. It blocks fumarate, a molecule that helps cells produce energy. Without fumarate, cancer cells struggle even more to survive. By combining these two effects, LCL768 attacks tumors on multiple fronts, causing their energy production to collapse and stopping head and neck tumor growth. Laboratory tests in both mouse models and lab-grown human tumors showed clear signs that the drug successfully triggered mitophagy and slowed tumor growth. When researchers added fumarate back to the cells, the tumors quickly began to grow again, confirming that the drug’s effects were directly linked to energy disruption.
What makes this approach unusual is its precision. Traditional chemotherapy and radiation often harm healthy cells along with cancer cells, which leads to side effects such as fatigue, nausea, and tissue damage. LCL768 targets processes that cancer cells rely on more heavily than normal cells, leaving healthy tissue largely unaffected. This means it may offer an effective treatment while minimizing the harmful impacts that often accompany cancer therapy.
Early results suggest that the method could provide new options for cancers that are difficult to treat with standard approaches. Low ceramide levels are common in many types of cancer, so boosting ceramide inside tumor cells could become a strategy beyond head and neck cancers. By focusing on the way cancer cells manage energy and stress, scientists hope to create a new class of drugs that are both effective and safer for patients.
While LCL768 has not yet been tested in humans, the research team is moving toward clinical trials. Their goal is to see whether this energy-targeting approach can become a real option for patients who currently have few alternatives. The early findings show promise for slowing tumor growth and potentially improving outcomes without the severe side effects associated with current therapies. Researchers say that exploiting cancer cells’ reliance on mitochondria and metabolism could open a new chapter in cancer treatment, giving hope to patients with tumors that resist conventional care.
The discovery also highlights the growing focus on understanding cancer from the inside out. Rather than attacking cells indiscriminately, scientists are looking for weak points that tumors depend on to survive. Compounds like LCL768 demonstrate that targeting these vulnerabilities can shut down cancer growth while preserving healthy tissue. This approach may redefine how certain cancers are treated and inspire similar strategies across a range of aggressive tumors.
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New drug dismantles cancer cell powerhouse to halt tumor growth
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