(RxWiki News) One of the genes that helps to keep cancer from ever forming is the p53 gene. It works by blocking cancer cells from killing themselves. When p53 is messed up in some way, cancer cells go to town and wreak havoc.
A new substance - APR-246 – was shown in a small clinical trial to repair and reactivate damaged p53.
The restored gene was then able to shrink tumors in patients with advanced blood or prostate cancer.
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Researchers at Karolinska Institutet and Karolinska University Hospital worked with 22 patients in a phase I/II clinical trial.
The individuals received APR-246 by infusion for four days. Patient cancer cells were analyzed before and after the treatment.
The scientists saw that the p53 gene was working at some level and that this activity inspired the cancer cells to commit suicide.
Of the ten patients who could be examined to see how their cancer responded, two showed signs of tumor shrinkage.
The trial was actually designed to see how well the substance was tolerated – not to measure its clinical effects.
The main adverse effects were tiredness, headache, nausea and confusion, all of which were temporary. So the study found that APR-246 is well tolerated.
dailyRx News reached out to lead investigator, Sören Lehmann, to learn more about the types of cancer involved in the research.
“The patients had acute myeloid leukemia, non-Hodgkin lymphomas, myeloma and prostate cancer,” he told us in an email.
Professor Lehmann said that the company supporting this research – Aprea AB – plans to test APR-246 in ovarian cancer, but not blood cancers.
“Personally, I think the biggest hope with this drug is in combination with chemotherapy,” he told dailyRx News.
The p53 gene is altered in about 50 percent of all tumors, so this compound may be an important find.
"In theory, a drug that restores p53 function should be effective against many different kinds of cancer, provided that the individual tumor contains defective p53," said study team member, Professor Klas Wiman, in an email. "We should keep in mind, however, that tumors are very complex."
This study appeared in the September issue of the Journal of Clinical Oncology.
Professor Wiman and his colleagues developed APR-246. The study was conducted in association with Aprea AB. Professor Wiman is a co-founder, shareholder and board member of Aprea.