Scientists could be within five years of discovering a cure for sickle cell disease, the head of the National Institutes of Health declared at a recent Capitol Hill event.
“Not just treat it but cure it,” which Francis S. Collins said would mark the first-ever cure of a molecular disease. “I think we can do that, and we should not rest until we do.”
What was once a fatal childhood disease is now a chronic condition, thanks to previous advances in medical research. But Collins thinks they can still do better. Sickle cell disease affects approximately 100,000 U.S. patients, disproportionately impacting African Americans, who continue to experience severe pain and hospitalization.
Some people would prefer the NIH director shy away from using the word “cure,” Collins said at the Sept. 13 reception for the Rally for Medical Research. They would prefer he use language about converting acute and serious conditions into chronic diseases. “But I’m still bullish about using the cure word,” the NIH director said, “because look at some of the things that are possible.”
(Personally, I’m a little grateful I never had to fit the “21st Century Converting-Acute-and-Serious-Conditions-Into-Chronic-Diseases” Law into the many, many stories about 21st Century Cures .)
Sickle cell disease is an inherited red blood cell disorder that turns the protein hemoglobin from its standard, flexible disc-shape into stiff, crescent-cell rods. These rods stop blood flow, thereby preventing oxygen from reaching nearby tissues and causing severe pain attacks. With advances in genetic sequencing and gene editing, Collins said researchers should be able to correct the sequence and re-infuse that into patients so they can have a normal blood flow, which means the patients would no longer have sickle cell and wouldn’t pass it to future generations.
“We should be—and I’m bold enough to say this—within five years of curing the first molecular disease,” he said.
It’s a bold statement indeed. But remember that Dr. Collins not only led the completion of the Human Genome Project, he also managed to convince President Donald J. Trump that he should keep the job President Barack Obama gave him back in 2009 to lead the NIH. And after telling Congress for years that flat budgets mean less NIH-funded research projects, aka fewer advances moving forward, the agency is on track to get a $2 billion increase from Congress—for the third year in a row.
“With no new money, we just simply have decided in a bipartisan way that we are going to prioritize what needed to be done here. We decided that medical research and NIH-driven research would be a priority,” Sen. Roy Blunt (R-Mo.) said at the reception. Blunt is the chairman of the appropriations panel that oversees health programs, including NIH funding.
Curing sickle cell disease was just one example of what Collins said this money could do.
I’m going to end this post by awkwardly and shamelessly plugging my Instagram account @MedResJourno, where you can see pictures from the rally.
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