The study involved 254 people with brain tumors and 238 people with no cancers. All those with tumors had glioblastoma multiforme, the most common type of brain cancer. People with this type of tumor survive an average of 12 to 15 months.
Through blood samples, researchers looked at the tumor suppressor TP53 gene. This gene acts as a tumor suppressor and is involved in preventing cancer.
People younger than 45 with brain tumors were more likely to have the Pro/Pro variant of the gene than older people with brain tumors or the healthy participants. A total of 20.6 percent of the young people with brain tumors had the gene variant, compared to 6.4 percent of the older people with brain tumors and 5.9 percent of the healthy participants.
"Eventually we may be able to use this knowledge to help identify people who have a higher risk of developing brain tumors at an early age. However the risk of this population remains low, even multiplied by three or four as shown here, because these brain tumors (glioblastomas) are infrequent in young people," said study author Marc Sanson, MD, PhD, of the French National Institute of Health and Medical Research (INSERM) in Paris, France.
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Two study subjects were recruited. (The number reduced from six study subjects following severe adverse events in an unrelated gene therapy trial taking place elsewhere, according to Evans.) Both subjects were postmenopausal females under the age of 75 with a diagnosis of advanced rheumatoid arthritis. After tissue was removed from the subjects' knuckle joints, a harmless retrovirus was inserted into the tissue cells, in order to serve as a "vector" to transport the gene into the patients' joints. After being placed in culture to grow and replicate, the cells were injected back into the afflicted joints.
After four weeks, patients reported reduced pain and swelling, according to Evans. "In one of the two subjects, these effects were dramatic, and the gene-treated joints remained pain-free even though other joints experience flares." Subsequent laboratory tests showed that tissues removed from the subject's joint tissue synthesized lower amounts of disease-related proteins, confirming that the reduction in pain and swelling resulted from the actions of the implanted gene.
"Existing treatments for rheumatoid arthritis are costly and need to be administered regularly," says Evans, adding that in addition to risk of side effects, not all patients respond well. "This paper provides us with the first real evidence that painful symptoms can indeed be lessened through gene therapy."
Ongoing work will focus on the use of gene therapy for the treatment of osteoarthritis, as well as rheumatoid arthritis.
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