Clinical trials are a type of controlled experiment carried out in clinical research. It’s worth stating how clinical trials differ from other kinds of experiments and studies with human subjects, such as surveys, case studies, and laboratory research. Clinical trials are labeled ‘controlled’ because they test a hypothesis about the effects of an intervention on people by comparing results between those that were given the treatment being tested and those that did not. Clinical trials are conducted to determine the safety and effectiveness of new drugs, vaccines, other therapies, and diagnostic tests. Here are five of the best medical discoveries through clinical trials.
1. A Diabetes Drug for Parkinson’s Disease
Parkinson’s Disease (PD) is one of the most common neurodegenerative disorders. It’s characterized by the loss of dopamine-producing neurons in the substantia nigra, leading to several symptoms, including tremors, rigidity, slow movement, and muscular stiffness. A drug called ropinirole, a common medicine used to treat PD symptoms, was tested in clinical trials conducted with people with Parkinson’s Disease. It was found that the drug significantly reduced tremors and slowness of movement in people with Parkinson’s Disease. Although ropinirole had no significant effect on rigidity or symptoms of balance and gait, it did help to improve motor function for heart rate instability, one of the most common complications of untreated PD.
2. An Anti-Depressant for Migraine Treatment
A new anti-depressant was studied in clinical trials for use as a treatment for migraine pain in adults. This is the first anti-depressant approved for adults with the disorder. There were two trials compared with a placebo. The first trial showed that people treated with the drug had a significantly greater reduction in depression symptoms than those given a placebo. In addition, this trial created evidence suggesting that anti-depressants effectively treat migraine pain and associated symptoms in adults. Clinical trial regulation guidelines require that people with serious illnesses, such as chronic migraines, be allowed to enroll in clinical trials.
3. ADC for Ovarian Cancer
A study demonstrated that an agent called Adcetris ideally targets cancer cells without damaging healthy, important cells. This was a first-of-its-kind study testing the agent in treatment regimens for people with heavily pretreated metastatic breast cancer or bulky relapsed ovarian cancer. This is the first combination approved for people with these advanced forms of cancer, who typically have limited treatment options. In addition, this is the first such therapy to show an overall survival benefit over single-agent chemotherapy in patients with metastatic breast cancer.
4. Crispr–CAS9 for Muscular Dystrophy
A new gene editing technique called Crispr-CAS9, which allows precise and accurate editing of targeted genes, was tested in clinical trials with people who have Duchenne muscular dystrophy caused by a genetic mutation that leads to muscle weakness. These trials were designed to test the safety and efficacy of a one-time treatment of this gene-editing technology. This treatment permanently modifies the DNA of cells so that they start producing functional dystrophin protein, which is needed to allow muscle cells to contract. This was the first time humans had used this technology anywhere in the world.
5. Cervical Cancer Screening in the Vaccinated
Women vaccinated against HPV, the virus that causes most cervical cancers, must still be screened because they’re at a higher risk of cervical cancer than unvaccinated women. A new study shows that combination cervical cancer screening is possible in this subgroup of vaccinated women and could allow them to reduce the number of procedures they need. Four trials tested different arm combinations with the Pap test and HPV test. Two trials showed no significant difference between screened women vaccinated against HPV and unvaccinated controls in both sensitivity and specificity for low-grade cell abnormalities, precancerous lesions, and high-grade lesions.
Clinical trials have led to the development of effective medical discoveries and innovations. They provide new treatments for diseases that were previously untreatable. They test new methods to diagnose and treat medical conditions, drugs, and other treatments. This decreases the risk of patients developing problems that could have been avoided. The bottom line is that clinical trials are important as they help to improve healthcare by providing vital information and testing new treatment options in people with a wide range of illnesses and conditions.