Leukemia girl heals 10 years later thanks to new therapy

Leukemia, girl heals 10 years later thanks to new therapy. The doctors had given her a few weeks to live

A 17-year-old given a few weeks to live while fighting leukemia at the age of six she recovered. Emily Weisskopfof Philipsburg, Pennsylvania, managed to cure the disease 10 years after the following one revolutionary therapy. “It’s really important to me to raise awareness about treatments like CAR-T cells,” he told People. “It’s a miracle I’m alive – and I’m so grateful.”

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The beginning of the disease

Emily started chemotherapy in 2010 but relapsed in October 2011 and was given only a 30% chance of survival. Emily’s leukemia progressed rapidly and became increasingly resistant to treatment. By February 2012, his health had deteriorated to the point where he was no longer eligible for a bone marrow transplant to treat the disease.

The new therapy

After being told that they had no other choice, her parents made her seek a second opinion at the Children’s Hospital of Philadelphia (CHOP). In doing so, Emily became the first pediatric patient in the world to have it CAR-T lymphocyte therapya treatment in which the patient’s cells are manipulated to attack cancer cells.

Emily was able to overcome all odds thanks to an innovative treatment made available just when she needed it. dr Stephan Grupp, the first director of the Susan S. and Stephen P. Kelly Center for Cancer Immunotherapy at CHOP, had just received approval to open the first phase 1 study of CAR T-cell therapy in pediatric patients with ALL. CAR-T cell therapy harnesses the power of a patient’s immune system by remodeling their T cells to attack proteins found on the surface of cancer cells.

How does it work

CAR-T cell therapy is a treatment in which a patient’s cells are designed to attack cancer cells. A specific immune cell – so-called T-cells – is taken from a patient’s blood. T lymphocytes help the body fight infection by scavenging for viruses and other pathogens before killing them. These cells are then modified in the laboratory to express a gene that codes for a specific receptor that binds to a protein on the patient’s cancer. Once these cells are reinfused into a patient’s blood, their immune system is “reprogrammed” to recognize and fight tumors. CAR-T therapy – chimeric T-cell antigen receptor – is therefore customized for each patient. It is suitable for people with advanced or worsening blood cancer who are not responding to treatment or who have come back. The US Food and Drug Administration approved two CAR-T cell therapies in 2017.