A breakthrough drug co-developed by the researchers from a university in Hong Kong could offer help to patients who are suffering from deadly brain cancer, the school revealed on Monday.
Approximately 200 people in Hong Kong were diagnosed with brain cancers each year, and about 20 of which were afflicted with a rare but aggressive type known as the Secondary Glioblastoma (sGMB), according to the South China Morning Post. Right now, treatments for sGMB include orally administered drugs and chemotherapy, but they have been proven to be ineffective treatments - with a mortality rate of about 100 percent.
Researchers from the Hong Kong University of Science and Technology (HKUST) announced they found out that a type of gene mutation causes the aggressive progression of the particular brain cancer. Wang Jiguang, a researcher and an assistant professor, said anyone who had sGMB could likely die, but their discovery shed some lights in which tumor cells can be tamed.
The research team has been working with Beijing Tiantan Hospital on sGBM patients since 2016. Among 14 percent of about 80 patients, the team identified a gene alteration called METex14 mutations. A protein gene in the brain experiences an increase in activity because of this mutation, activating the sGMB cells and eventually leading to the tumor's growth in the brain.
The discovery prompted researchers to arrange the patients to undergo a new kind of treatment known as the PLB-1001. The team prescribed patients to take a daily dosage of the oral drug ranging from 50 to 300 milligrams in three months.
Wang said they saw a connection between the PLB-1001 drug and those who are experiencing METex14 mutation. The drug was used as a stand-alone drug, where they saw the tumor's shrinkage within three months in selected patients.
The team studied 18 patients, wherein two of them reportedly experience a reduction in their tumors and no recurrent symptoms for as long as three months. Seven patients were also said to remain stable for 12 weeks. Meanwhile, researchers said patients would surely die within 10 years without the drug.
The study pointed out that the drug molecule could penetrate to the blood barrier, reaching the brain's tumor. The researchers considered their discovery, as well as the results, a breakthrough. Wang also noted those involved in their study were chemo-resistant patients who have not responded to any treatments, so their positive response to the drug is already a quite success.
The PLB-1001 FDA approval is still pending, and Wang said further studies are needed to find out whether the drug can be used in conjunction with other drugs for a longer lasting outcome.
