In our last post, we explored the different phases of cancer research. One of these phases—translational research—is sometimes referred to as the “Valley of Death.”
Translational research got this reputation because the process of translating early discoveries into effective treatments for patients is time-consuming, costly, and often unsuccessful. A discovery in basic science—for instance, a new way of monitoring how a certain tumor spreads through the body—must be followed by years of additional work before it becomes a treatment. One recent article estimated that the rate of success in translational science is less than 1%. This means that if 5,000-10,000 compounds are tested, only about 5 will end up in Phase 1 studies, and only a single drug is likely to survive to become approved by the FDA.
If we follow the tumor discovery down the drug pipeline, we begin with researchers trying to figure out what proteins, enzymes, or other targets will accompany the tumor they can now track, and which of them might be susceptible to treatment. Perhaps after much testing, researchers discover that a single protein is instrumental to the growth of the tumor. The next step is to find compounds that will inhibit this protein, thus inhibiting the spread of the tumor. They might test hundreds or even thousands of compounds to see which ones work to inhibit this protein. Then, they need a good model of the tumor to test these potential compounds. Once they have narrowed down the compounds to the hundred or more most effective drugs, they will test them on animal models. Both tumor models and animal models take time and expertise to develop to ensure they are accurate and behave like a tumor in a human.
The handful of potential treatments that make it through these initial phases and through pre-clinical testing may go on to Phase I studies in humans, the next stage of the research pipeline. If researchers are incredibly lucky, one of these treatments will be effective in treating the disease and patients will be able to tolerate it. After several more rounds of clinical trials, the drug can move on to the expensive and lengthy process of receiving FDA approval for wider use.
Although translational research is a crucial step for discovering new cancer treatments, it is an under-funded research area, in part because the risk of failure is so high. While a discovery in basic science has the potential to impact hundreds of disease areas, translational research is the crucial bridge between the laboratory and improving outcomes for patients today. In addition, because early research is so often funded by government and clinical trials are often funded by pharmaceutical companies, it can be difficult to move between these different environments and funding models. You can find more information about translational research here, here, or here.