UCLA Case No. 2019-281
Summary
UCLA researchers in the department of Dentistry have developed a novel anti-cancer treatment that directly targets enzyme expression necessary to sustain cancer growth.
Background
The incidences of cancer diagnosis in the United States is reported to grow continually in the next decade. While there are a growing number of proposed cancer therapies, limited successes have so far been found. The typical widespread use of chemotherapeutic treatments target cancer cells with great efficacy, but also have off target effects to healthy cells: causing many of the well-known side effects of such therapies. While a large emphasis has been stressed for the need of more on-target small molecules, development in this field is slow moving and have been focused on cancer metabolism or cell surface receptors to control signaling pathways. However, these signaling mitigation agents have limited potency and require intensive dosing regimens.
The advent of oncogene discovery and its growing evidence in literature has turned great interest into directly effecting genes that control the machinery that cancer cells use to survive. However, many of these oncogenes are difficult to target and control basic cellular function in otherwise healthy cells. Therefore, the identification of oncogenes that are necessary in cancer cells but otherwise unneeded in healthy cells is a contentious field. Beyond this, the delivery of agents to directly affect cancer genes without causing other unintended gene mutations is quite difficult. Therefore, the identification of a necessary cancer gene and a delivery method for its knockdown could be an efficient means to future cancer therapy.
Innovation
Dr. Hu at UCLA has developed a novel siRNA sequence that targets gene sequences that control PLOD3 enzyme expression. PLOD3 has been shown to be highly overexpressed in malignant cancer cells and its knockdown could lead to an efficient way to inhibit cancer growth. Preliminary research with the siRNA sequences packaged in a lentivirus have shown that its transfection into a variety of human based cell lines limits growth. When these cells were introduced in-vivo, tumor growth was significantly inhibited. These initial discoveries mark a new frontier into cancer therapy, where oncogenes could be directly inhibited to provide a better and more effective cancer therapy than traditional routes.
Applications
▶ The use of a single time transfection to effect cancerous subpopulation of cells
Advantages
▶ The only technology of its kind that directly effects oncogenes
State Of Development
The invention has been tested both in-vitro and in-vivo to selectively inhibit tumor growth while limiting off target effects; proposing an effective treatment without major side-effects.