Clinical Trials in Oncology: An Option for Innovative Treatment

"Receiving treatment within the framework of a clinical trial is the preferred option for patients fighting advanced stages of cancer. It also offers the best chance for achieving breakthroughs in cancer treatment and medications"

Treatment within the framework of clinical trials is the preferred and recommended treatment alternative for many cancer patients in advanced stages of the disease.

Often the opportunity to receive advanced and innovative treatment available only via clinical trials is a patient's only chance of attaining improved results in their condition, especially in cases with widespread metastases. Furthermore, breakthroughs in cancer research and treatment are made possible only as a direct result of the conduction of clinical trials that attract many participants.

Cancer is a serious disease. In many cases, when a cancerous tumor is not fully removed, the disease will lead to the patient’s death. In cases where the cancer could not be fully excised, the patient is generally given drug therapy that can prolong and improve the quality of life.

However, drug therapy does not usually lead to complete recovery. It is for this reason that each year billions of dollars are invested in the research and development of new drug treatments for cancer.

The only way to assess the efficacy of a new cancer medication is through clinical trials. Clinical trials are in a essence a win-win situation for both the cancer patient and the pharmaceutical company. The patients have access to innovative treatments that would otherwise be unavailable, and the drug companies are given a chance to prove the efficacy of their newest developments.

Clinical trials involving oncology patients are generally designed to allow patients to receive the normally prescribed treatment for their disease in addition to the trial drug. This is done to ensure that participation in trials will only increase patients’ chances of survival and recovery. In many trials where the clinical drug's efficacy is proven, the patients’ condition can improve significantly.

Unfortunately, many patients feel uncomfortable at the thought of participating in and receiving treatment through clinical trials. The notion of serving as guinea pigs in an experiment causes patients to reject the option offhand. This fear is not without basis. In the past, the lack of clear guidelines for the conduction of clinical trials in humans did lead to some cases in which patients receiving experimental drugs suffered unnecessarily, and instead of improving, their medical conditions became more severe.

This unfortunate situation resulted in an improvement over the last few decades. Consequently, international medical committees have been established and a clear-cut policy has been formulated to deal with the issue of clinical trials in humans. These committees operate at the hospital level (Helsinki Committee) and on a national level, in order to protect the participating patients. Nowadays, every trial is subject to the committee’s rigorous review, ensuring that the high standards of performance from such trials are fully met, in terms of quality and transparency in regard to treatment, medications and patient monitoring.

Deliberations held by the world’s top experts have led to the formulation of new operative standards pertaining to all physicians conducting medical trials. This standard is called Good Clinical Practice (GCP). It includes detailed guidelines regarding the physician’s and the medical system’s treatment of all participating patients. The main goal of these guidelines is to ensure the patient’s safety and well-being within the framework of the clinical trial.

The GCP's detailed guidelines encompasses the entire realm of medical activity, including:

Obtaining the patient’s informed consent.

Compliance with the schedule of the clinical trial.

Guidelines to be followed in case of the onset of mild or severe side effects.

Assessing the patient’s response to treatment.

Numerous responsibilities related to patient treatment and monitoring, required reporting to the various ethical committees, directives regarding analysis of findings, as well as transparency and exposure to inspection.

Consequently, in cancer treatment centres that conduct clinical trials using GCP guidelines, there has been a vast improvement in the level of care and treatment. So much so, that in fact, patients not undergoing clinical trials have also benefited from the change. This is due to the fact that the doctors who are being held to the high standards mandated for clinical trials are implementing these same standards with the rest of their patients.

Clinical trials are categorized according to stages, as follows:

First stage clinical trials involve the administration of a newly developed drug for the first time in human patients. First stage trials are generally meant for terminal patients who are still able to function and who feel relatively well; however, they have no other viable and effective treatment options available. The only alternative for patients who are offered participation in first stage clinical trials is palliative care. There are very strict rules for carrying out the treatment protocol in this option with the focus on protecting the patient from any possible side effects. The main goal of this trial stage is to identify the treatment’s side effects. For the patient, this is an opportunity to benefit from the most recently developed treatment, which may even halt the progress of the disease.

Second stage clinical trials involve medications that have been proven to be safe in the first stage, and their efficacy is now being tested on patients with a particular type of cancer. Generally, first and second stage trials are designed without the need for a control group.

Third stage clinical trials compare the efficacy of the trial medication to conventional treatment at a certain stage of the disease. Participants in third stage trials (the most common type of trial) are assigned to one of two groups: either to the group receiving the conventional treatment or to the group receiving the experimental treatment.

In many cases, patients in the experimental group continue to receive the conventional medication. Third stage clinical trials are a double-blinded study, in which neither the patient nor the treating physician knows to which group the patient has been assigned. This is in order to avoid any bias effects such as the placebo effect.

If and when a certain drug is proven to be more effective than the conventional treatment it is submitted for a review process. If the trial results are corroborated, the drug will become the standard treatment for the specific disease treated in the trial stage.

The enormous progress in recent years in the treatment of various types of cancer would not have been possible were it not for these processes and the active participation of thousands of cancer patients in clinical trials. Trials that determined which new drugs were indeed effective in the battle against this disease.

Dr. R. Berger is a senior oncologist