This year’s World Sickle Cell Day on June 19 may mark the first time that increased awareness is being met with real advances in battling Sickle Cell Disease.
In addition to national publicity on the TV show “60 Minutes” in March, there are 40 drugs and treatments being tested right now, says Kevin Wake, a volunteer with the Uriel E. Owens Sickle Cell Disease Association of the Midwest and a Sickle Cell patient.
The U.S. Food and Drug Administration has announced that it’s “fast-tracking” tests of a take-once-daily pill called IMR-687, the National Institutes for Health are accelerating gene therapy tests, and Howard University is hosting a symposium on June 19 to address new innovations.
“Sickle cell disease results from a single genetic mutation that causes a person’s red blood cells to form an abnormal, sickle shape,” the NIH website, nih.gov, says. “These sickled cells can clog the blood vessels and deprive cells of oxygen. In turn, this lack of oxygen wreaks havoc on the body, damaging organs, causing severe pain, and potentially leading to premature death.”
“I think there’s a lot of misunderstanding about the disease,” said Wake, who advocated for patients with members of Congress last year during Rare Disease Week. “You can’t catch it; you’re born with it. You can also carry the trait genetically and not have the disease, but can pass it to your children.”
The child of two trait carriers has a 25% chance of having the disease.
Sickle Cell is classified as a “rare” disease, but it afflicts 100,000 Americans and 4.4 million people worldwide, and though many patients are Sub-Saharan Africans, it’s not just a “Black” disease, Wake said. Other ethnicities affected by it include Hispanics, Iranians, East Indians and Greeks.
Sickle Cell is now the most common genetic disease in France and Jamaica. Outside the U.S., life expectancy for Sickle Cell patients is about 5 years old due to lack of access to treatment, Wake said. It’s 40 to 60 years here.
The great part about the new treatment/cure initiatives, he said, is that the gene-editing and stem-cell technologies being pioneered for Sickle Cell can be translated for use with other diseases.
To support patients, people should donate blood regularly because it’s always needed for treatments and there’s currently a shortage (see Sidebar). Also, join the national registry to donate stem cells for Sickle Cell and blood-cancer patients at join.bethematch.org/CURE44.