The breakthrough

12/14/2023, 6 p.m.
A long-awaited treatment for sickle cell anemia is highlighted in this week’s Free Press.

A long-awaited treatment for sickle cell anemia is highlighted in this week’s Free Press.

That’s good news we all can use and possibly take our minds off of City jail investigations, lawsuits against the Richmond school board, an ex-president trying to weasel his way back into the White House, and too many wars to count (more than 45 in the Middle East and North Africa).

Thus, bravo to the dogged determination of medical and research scientists for the FDA’s approval of two milestone treatments, Casgevy and Lyfgenia. The treatments represent the first cell-based gene therapies for sickle cell disease (SCD) in patients 12 years and older.

One of these therapies, Casgevy, is the first FDA-approved treatment to utilize a type of novel genome editing technology, signaling an innovative advancement in the field of gene therapy, the FDA stated Dec. 8. The announcement stirred excitement in Black communities everywhere.


Sickle cell disease is a group of inherited blood disorders affecting approximately 100,000 people in the U.S. It is most common in African-Americans and, while less prevalent, also affects Hispanic Americans.

The primary problem in sickle cell disease is a mutation in hemoglobin, a protein found in red blood cells that delivers oxygen to the body’s tissues, according to the FDA. This mutation causes red blood cells to develop a crescent or “sickle” shape. These sickled red blood cells restrict the flow in blood vessels and limit oxygen delivery to the body’s tissues, leading to severe pain and organ damage called vaso-occlusive events (VOEs) or vaso-occlusive crises (VOCs). The recurrence of these events or crises can lead to life-threatening disabilities and/or early death.

“Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we are excited to advance the field especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today,” said Nicole Verdun, M.D., director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research. “Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited.”

Thus, in Richmond, the FDA announcement is especially welcome where the late LaVerne Amelia Wingo Cooper devoted her life to trying to find cures for the disease. Mrs. Wingo Cooper, who died last September at age 92, was a clinical researcher at the Medical College of Virginia whose work focused on sickle cell anemia. Physician Robert B. Scott Jr. and Richmond social worker Florence Neal Cooper Smith also have worked for decades in leading the fight to raise awareness of the disorder and improve treatment through a research center at MCV, Virginia Commonwealth University’s medical arm.

Others who’ve championed awareness of sickle cell anemia and ongoing research for a cure include Dr. Wally R. Smith, who currently leads the adult sickle cell research and treatment clinic at VCU’s medical school and hospital. There’s also Allison Coles-Johnson, a retired nurse who co-chairs the Richmond-area nonprofit, Finding A Cure for Sickle Cell Anemia, describes the new therapies as an “excellent start,” but cautions that there is a need to continue “research so that therapies like this can become more accessible and more attainable” for those with the disease.

Dr. Smith acknowledged that the cost of the new treatment may be a barrier. According to sickle cell advocates, these new one-time, single-dose therapies are expected to cost at least $1.2 million, and possibly more.

Cost is already a barrier to existing sickle cell medications, our Free Press report notes. For example, the cost of Oxbryta, a Phizer medication to increase red blood cell production, has jumped in cost from $2,200 a month five years ago to nearly $12,000 a month currently, according to Sickle Cell-Virginia.

So while the new treatments have been approved, there is no rest for the weary as Dr. Smith, Ms. Coles-Johnson, and others continue to tackle barriers encountered by many sickle cell patients.

What can you do? Learn more about this daunting disease that has claimed the lives of far too many of our friends and loved ones. Visit VCU’s Sickle Cell Disease Program website

(https://intmed.vcu.edu/about/institutes/sickle-cell/), sign up and participate in one of the charitable fundraisers, runs and walks that take place to raise awareness about and money to fight the disease. Become a volunteer for a sickle cell-related event. Contribute to scholarship programs for college students in STEM or social work disciplines.