A groundbreaking technique that uses a disabled form of the virus that causes AIDS has successfully treated a 6-year old with leukemia. The research technique essentially reprograms the patient's immune system to kill cancer cells giving the patient's own immune system the lasting ability to fight cancer.
Whilst in its infancy, the pioneering research -- carried out at the Children's Hospital of Philadelphia -- has opened up a potentially life-saving avenue of therapy and points to a first in translational "bench-to-bedside" experimental treatment that could lead to less toxicity and longer, healthier, life spans for survivors.
The experimental treatment was the focus of a short film that formed part of a GE Focus Forward / cinelan-sponsored Vimeo series of 3 minute documentaries on "big ideas." The illogicality of this research gave filmmaker Ross Kauffman the perfect subject matter to fulfill the creative brief: tell a story about an innovator or a world-changing idea and do it in about 180 seconds.
At the center of the short film was Emma Whitehead, a then 6-year old who was suffering from leukemia. She had relapsed twice after previous chemotherapy. Desperately running out of options, her parents agreed for Emma to undergo an experimental treatment at the Children's Hospital of Philadelphia, one that had never before been attempted in a child, or in anyone with the type of leukemia she had.
"Patients that entered this clinical trial have absolutely no other options left to them," said Stephan A Grupp, MD, PhD, Children's Hospital of Philadelphia. These are patients that are destined to die from their disease in a short amount of time. Emma was a patient that had had her leukemia come back twice. The parents were essentially looking for a miracle."
Doctors began by removing millions of the patient's T-cells -- a type of white blood cell -- and introducing new genes that empowered the T-cells to kill cancer cells. The method used a deactivated form of HIV because of its ability to carry genetic material into T-cells. The new genes then instructed the T-cells to attack B-cells, a normal part of the immune system that turn malignant in leukemia cases.
The transformed T-cells -- named chimeric antigen receptor cells -- are then introduced into the patient's veins, hopefully beginning to multiply and destroy the cancer. The T-cells target a protein called CD-19 that is located on the surface of most B-cells, whether they are healthy or malignant.
"Emma was given her T-cell treatment and within a few days she was very sick," said David L Porter, MD, University of Pennsylvania. "She had breathing and blood pressure difficulties. We knew that she could not have gotten any sicker without actually dying."
"But then a remarkable thing happened," said Carl H June, MD, University of Pennsylvania. "The T-cells were growing and were starting to fight the cancer. Within hours Emma's fever disappeared."
While the physicians have acknowledged that this treatment is in the very early stages of development and is not fully understood, news of its success has great potential. The idea that a gathering of activities which seek to translate the science of gene discovery, gene transfer, and functional genomics could be used as gene-targeted therapies is one that has been bandied about for some time. However, the applications of this science in real-life situations have not made as much progress as scientists struggle to assign a universal definition of it.
Know more about GE Healthcare products
Discover the latest Business News, Sensex, and Nifty updates. Obtain Personal Finance insights, tax queries, and expert opinions on Moneycontrol or download the Moneycontrol App to stay updated!
