Why clinical trials can be your lifesaver

Tuesday, September 9, 2014
By, Eric

by Erad le Beau de Hemricourt, MD

In the recent years medicine has been advancing at a tremendous speed, with new discoveries every day. Some of these discoveries are in the form of potential drugs for devastating diseases such as cancer. There might thus be various options to treat a given disease. The number of these new therapies –  including the ones in the pipeline – is so vast that it is hard for a single physician to keep track of all of them.

In other words, be it cancer, hypertension, diabetes or cardiovascular diseases, doctors may face hard choices given various different therapeutic options. This was not always the case, but nowadays it can be very tricky for a medical specialist to decide on the right drug to treat an ailment, especially when the drugs that are about to finalize their test phase are also thrown into the mix. This test phase is part of the overall methodology to investigate whether newly discovered medication really works, what the optimal dose should be and what the possible interactions with other medications are. Test phases are also referred to as “clinical trials”. In clinical trials, newly discovered treatments will be tested in various phases, i.e., phases I, II, III and IV. Only when a potential new molecule successfully passes a phase, can it move on to the next one.

Cancer is a complex disease, and there is a wide range of new developments in this field. When a cancer is detected, depending on the type and stage of the tumor, doctors can choose among the options of surgery, radiation therapy, chemotherapy, hormonotherapy, immunotherapy or targeted therapy. And even when the choices are narrowed down to chemical drugs, several therapeutic options remain based on certain questions such as the following: Which hormone should be selected for a specific tumor? Which new immunoglobulin should be used to treat a specific type of cancer? So you can imagine that doctors can encounter some difficulties in selecting the best treatment for you. That is why, especially in oncology, doctors have to follow certain guidelines and protocols, and decide on the best option during their weekly meetings with other medical specialists. Here their goal is clear: gathering enough information to treat their patients in the best possible way by exchanging experiences and knowledge.

But what many patients do not know is that these guidelines vary not only across  countries but also across hospitals within each country. Why? First of all, there are many different treatments for the same kind of tumor. Secondly, there is no crystal ball telling us which treatments will work and which others won’t on a case-by-case basis; doctors still use the old system of trial and error for certain types of cancer. On the other hand, there are very strict international guidelines to be followed for drugs tested in clinical trials. These guidelines are in place to make sure that all the results are as accurate and reliable as possible for regulators’ authorization to release the drug on the market, after showing some therapeutic efficacy.

Considering the present frontier of drug research for cancer, the good news is that we have entered a more genomic era quite recently. We are indeed just starting to have a direct insight into the complex DNA disruptions that eventually lead to tumors. We can thus fight tumors by understanding their genetic mechanisms. Sequencing cancer genome is complex and costs a lot of money. That’s why the process might take 10 to 20 years before we get the whole “picture”. Yet we do not need to wait until 2030 to see any potential results, since they are already showing up. In this context, just consider some recent cancer drugs targeting very specific genetic mechanisms, such as Gleevec (leukemia), Herceptin (breast cancer) or Vemurafenib (melanoma).

These new drugs have already saved thousands of lives. And this is just the beginning! According to the Food and Drug Administration, there were 19 new anticancer products approved in 2012, followed by 12 in 2013, and 5 in the first half of 2014. These new targeted agents are just the tip of the iceberg as thousands of potential new anticancer molecules are under current examination. The number of targeted agents to be tested will increase significantly within the next few years. All of these agents will be tested in clinical trials to prove their efficacy.

Then, we are faced with the following big question: how can you, as a patient, get a chance to try one of these new products? The answer lies in selecting the right clinical trial to participate in! Currently, there are around 30,000 clinical trials in oncology alone throughout the world, so being part of the right clinical trial can also be tricky due to specific inclusion/exclusion criteria. You need to invest some time reading about all the relevant clinical trials, and discussing them with people around you, i.e., other patients, doctors and especially cancer specialists. After having enough information, you need to decide whether or not to participate in a given trial.  Going through a clinical trial will provide you with a thorough medical screening and a close follow-up, which in themselves can be beneficial. As some of the drugs are experimental, there might, of course, be some unknown side effects that will be closely monitored by the researchers. On the other hand, the drug you test may indeed be a lifesaver by stabilizing or curing your cancer.



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