From Treating Symptoms to Treating the Source

Medical advances have resulted in more therapies that can have a real effect on the cause of disease.

For generations, treatment for many illnesses has been limited to easing symptoms, with the hope that it will also control or cure the ailment. With something as common as influenza, for example, treatments traditionally focused on reducing fever and providing comfort for sore throats and muscle aches and pains. Many over-the-counter flu remedies still aim to relieve symptoms. But after the influenza virus was isolated in the 1930s, therapies shifted to preventing infection through the development of targeted vaccines and shortening illness duration with antiviral medications, which attack the source of the problem, the flu virus itself.

Better diagnostics that correctly identify disease, treatments that improve health and the promotion of lifestyle changes that increase life expectancy are all important advancements in medicine. But in the case of treatments, a major sign of progress in recent decades was the shift from simply controlling the signs and symptoms of disease to addressing the underlying cause. Advances in scientific research led to a better understanding of the causes of disease, resulting in the identification and development of more appropriate therapies for a variety of illnesses.

In the blood cancer myeloma, for example, the past two decades have seen disease-modifying treatments become the standard. These novel treatments have supplanted older therapies that focused mainly on treating only the effects of the disease, such as anemia and infections. Now, researchers are looking to identify the best treatments at the best time, to achieve the best outcomes. The goal is to advance from long-term remission, which patients can now achieve, to a true cure. To do so, doctors need to be able to follow how myeloma cells react to each treatment over the course of a patient’s treatment path.

Advances in scientific research led to a better understanding of the causes of disease, resulting in the identification and development of more appropriate therapies for a variety of illnesses

“We need to consider the treatment with the right patient, at the right time—in this case, early on—and measure all the way along what we are accomplishing, to make sure that we are continuing to knock any residual disease down to the lowest possible level,” Dr. Brian G.M. Durie, MD, chairman and co-founder of the International Myeloma Foundation, when he spoke at an educational myeloma workshop held in tandem with the American Society of Hematology’s annual meeting in December 2013.

Looking to modify the cause of disease has helped with the treatment of many serious illnesses. It has been discovered, for instance, that viruses may cause some forms of cancer, according to several studies. The root cause of cancer, which occurs when mutations cause cells to divide uncontrollably, has mystified scientists for decades. While mutations can occur naturally over time, scientists now think that viruses, which insert themselves into DNA to survive and replicate, make mutations more common.

“Viruses don’t set out to cause cancer, but their replication uses all the same functions,” Philip Stevenson, a researcher at the University of Cambridge, told Live Science. “So they’re kind of ideal agents for causing cancer.” Stemming from this type of research has been the human papillomavirus (HPV) vaccine, which is now used to help prevent cervical cancer. Finding a virus responsible for other cancers may lead to similar innovations.

The treatment of the blood cancer myelodysplastic syndromes (MDS) has also benefitted from the identification of the underlying cause of disease. MDS is a form of cancer in which healthy blood cells are lacking and abnormal cells are instead produced. Supportive care to alleviate symptoms such as anemia and bleeding is still a mainstay of MDS treatment. Recent advances, however, have identified new approaches that actually modify the disease, by eliminating cancerous cells and promoting the growth of healthy blood cells. These treatment strategies include novel therapies for patients whose MDS is associated with a missing piece of chromosome 5. Novel therapies have also been developed to treat an advanced form of MDS known as acute myeloid leukemia.

Cancer isn’t the only disease affected by this shift. Psoriasis, a skin condition that results in scaly patches of skin that can significantly impact quality of life for the patient, was traditionally treated with just topical medications. The goals of treatment were relief of symptoms, including pain relief, and prevention. These were accomplished with lotions, creams, exercise, diet and medicines that suppress multiple parts of the immune system.

Now, though, times are changing. In the 1960s, researchers determined that psoriasis is caused by an immune system gone awry. As a result, close to 40 new therapies are in development for psoriasis, including injectables, oral compounds and topical treatments. Many of these compounds modify the disease by targeting specific members of the immune system that are now known to be involved in the development of psoriasis.

As researchers have learned more about how our cells work and how processes go wrong in disease, they’ve also learned how better to address those defects. As a result, patients have benefitted from new, more advanced treatment choices for a variety of diseases and conditions, including cancer, heart disease, diabetes and inflammatory diseases. Since treatments that target the disease are more likely to lead to better outcomes for patients and may slow or even cure disease, medical innovations such as these are helping patients around the world live longer, better lives.