"Why get involved?"
It's a question we routinely ask ourselves in many situations. On June 19, World Sickle Cell Awareness Day, I revisit this question in the context of the great need for people to get involved in the process of finding treatments for this painfully debilitating condition.
We've known about sickle cell disease for more than 100 years, yet there are still few medicines to help patients or address the underlying cause of the disease. It hasn't been for lack of effort.
Over the years, a number of new therapies have been tested. Some have failed because they weren't safe enough... or effective enough. But more than half have failed for a sadder reason—the developer of the medicine wasn't able to recruit enough patients to complete the clinical trials to demonstrate whether the was safe and effective. It's possible that a promising therapy for sickle cell disease has been left on the laboratory equivalent of the cutting room floor.
Sickle cell disease is a rare inherited disease affecting about 100,000 Americans - enough to fill Yankee Stadium, Madison Square Garden, and the Barclays Center—and still leave thousands of people waiting for seats. About five times a day, a child with sickle cell disease is born, the vast majority to African-American parents.
People with sickle cell disease have misshapen red blood cells that interfere with other types of blood cells and cause inflammation. This cascade of events can block the flow of blood through the body causing painful crises so intense in some cases that patients must be hospitalized. Other serious health consequences can include stroke, acute chest syndrome, joint damage, kidney issues, occasionally even heart problems.
At Pfizer, we are researching new therapeutic approaches for sickle cell disease, including medicines to manage symptoms of sickle cell disease as well as therapies to treat the underlying cause. One of these, a potential therapy to reduce the tendency of the misshapen blood cells to clump together, could help free patients from pain more quickly and allow them to leave the hospital sooner, at great relief to them and our overburdened healthcare system.
We want this medicine - and all of our medicines - to be tested rigorously to prove that they work and that they're safe. That's why participation in clinical trials is so vital. We need to learn from the experience of participants so we can show the results to the regulatory agencies who determine if a medicine can be approved to help patients.
Those who join clinical trials are the heroes—unsung heroes, really—of biomedical progress. Without them, progress simply would not be made.
The good news is that science is moving forward. But we cannot achieve success alone. The faster we can find qualified patients for clinical trials, the faster we can potentially bring an exciting new therapy to those who are literally crying out for them.
If you or someone you know is affected by sickle cell disease, use this opportunity to reflect on whether you can get involved in being a part of the solution.
Talk to your doctor about getting involved with a clinical trial to see if you might be able to participate. Together we can lift the burden of disease for these patients. For these patients and their families - every day is "sickle cell day."
Kevin W. Williams, M.D., J.D., M.P.H. is Chief Medical Officer, Rare Disease at Pfizer, Inc.