By Patrick Cox April 14, 2011 (a buy recommendation was issued September 2012 and a new report should be coming out anytime now) In this month’s issue, I have a very exciting company for you. Although the company is very early-stage, with no human clinical data yet, I believe the science is sound. This company has discovered a compound that modifies a well-studied cellular process implicated in a broad range of cancers. For many years, it has been a holy grail of cancer researchers. Tens, if not hundreds, of millions of dollars have been spent by pharmaceutical companies in the quest to find a therapy to hit this molecular target — but in vain. The tiny biotech we’re presenting this month, however, truly appears to have discovered the compound that does so successfully. Obviously, preclinical biotechnology innovators make for more speculative investments than those that have reached the stage of clinical trials. With greater risk, however, also comes the potential for greater reward. The biggest yields in pharmaceuticals come from picking winners before human data verify the safety and efficacy of a drug in humans. Of course, the flip side of that coin is the possibility that clinical trials will fail. Most investors and analysts, therefore, tend to rely solely on human clinical studies as the only means of judging a drug candidate. This is simply because most people lack the time, background or confidence to study and grasp the core scientific platforms of preclinical companies. This creates opportunities for investors who are willing to do their homework and buy into companies before clinical results drive share prices up. Fortunately, we’re not living in the 20th century anymore. With Google Scholar and a little patience, you can instantaneously access information that could be found only in large university libraries just a few decades ago. So I would like to tell you about a number of factors that indicate this company’s pipeline has transformational potential. Beyond the core science, which I’ll describe below, there is also the reputation and decades of experience of its founder and chief scientist. Additionally, strong preclinical data and outside interest lead me to believe the probability of success justifies adding this company to your watch list of transformational technologies. However, I encourage you to consider this technology carefully. P53, The Guardian Angel of the Genome Before addressing the company, though, I would like to talk about cancer and the “guardian angel gene.” Known to geneticists as TP53, this gene encodes the transcription factor p53. Lately, you’ve been hearing the term “transcription factor” in this publication quite a lot. There’s a reason for that. As the human genome and the proteins expressed by genes are understood, more and more breakthroughs will be enabled. When this protein was discovered in 1979, the tools did not exist to fully analyze and understand the TP53 gene or its protein transcription factor p53. The scientific community assumed, in fact, that p53 was causing cancers. Later research revealed a much more complex and interesting story. P53 is, in fact, a cancer-suppressing transcription factor, rather than a cancer-causing one. After discoveries revealed its role in preventing cancers, the journal Science named p53 “Molecule of the Year” for 1993. Additional discoveries are still being made about p53. This protein clearly operates as a transcription factor, controlling the conversion of DNA information to messenger RNA, or mRNA. It also, however, seems to act in a pharmacological manner. Regardless, p53 plays an extremely important role in guarding cell health. Specifically, p53 monitors the DNA and activates cellular gene repair processes when a cell sustains genetic damage. If genetic damage proves too extensive for repair, p53 triggers the cell’s built-in self-destruct process, apoptosis. For this reason, the p53 protein has been called the master watchman of the genome. As such, it prevents the mutations that turn healthy cells into cancer cells. There is an Achilles’ heel to this process, however. If the p53-encoding gene, TP53, itself receives damage, there is no mechanism to repair it as p53 repairs other genes. Its tumor-suppressing functionality is curtailed. Similarly, if some other factor interferes with p53's normal functioning, the TP53 gene can no longer prevent cancers. This is the reason that human papillomavirus (HPV) leads to an increased risk of cancer. When HPV infects a cell, it causes it to manufacture a protein that binds to p53, inactivating the master watchman. For some cancers, such as cervical cancers, HPV is believed responsible for 70% of cases. In more than half of all human cancers, p53 is suppressed by mutations of the TP53 gene itself. The other cancers directly inhibit p53's molecular signaling pathway. P53 suppression, therefore, is implicated in almost all cancers. If some molecule could restore p53's activity in cancer cells, it is believed that the cancers would be naturally and nontoxically eliminated. Such a molecule would be a powerful new oncology drug.