Disease Modifying Therapies

NOTE: Benefits and risks exist for ALL medications. Please consult your physician to discuss your options and help determine which treatment option(s) may be right for you.

Gene Therapy

Gene therapy involves altering the genetic coding inside your body's cells in an effort to treat or stop disease. Genes contain your DNA — the code that controls much of your body's form and function, from making you grow taller to regulating your body systems.

All babies in the womb and newborns have a certain version of hemoglobin called fetal hemoglobin. Even babies with sickle cell disease make normal red blood cells because fetal hemoglobin doesn't sickle. But shortly after birth, the body stops making fetal hemoglobin and starts making adult (A) hemoglobin. The reason this is critical for sickle-cell patients is that the gene mutation is what causes the body to make an adult hemoglobin variant, sickle (S) hemoglobin. Gene therapy in sickle cell works by flipping the body’s switch back to fetal hemoglobin, simultaneously increasing fetal hemoglobin, which does not sickle, and directly reducing sickling (S) hemoglobin. 

To perform gene therapy, a patient’s blood stem cells are collected and exposed to a vector containing instructions to knock down BCL11A (the gene mutation that causes SCD). The patient then receives chemotherapy in a process called conditioning, likened to plowing a field to make room for new seeds. Finally, the gene-modified cells are given back via intravenous infusion.

Gene therapy (or gene editing) does not require a donor and is currently in various stages of clinical trials. 

Blood Donor Diversity

If something is FOR US, it must be WITH US

WE ARE THE US WE’VE BEEN WAITING FOR!

Since nearly the beginning, there has been a large gap between the number of individuals needing blood or bone marrow and the number of folks donating it. This gap is even wider when we are talking about people of African descent donating or having access to these life-saving donated products. In 2020, shortly after the start of the pandemic, this disparity was glaringly obvious as the shortage of blood donors created a disturbing ethical debate. Which patients get blood and which patients do not! Thankfully, the Red Cross, Be The Match, and others have stepped up to ensure that blood and marrow remains readily available to marginalized communities and chronically ill patients! To do this, they also had to make donor diversity a top priority.

So why do Black donors matter? Every ethnic group has genetic

similarities that increase compatibility markers and when medical complications such as acute chest syndrome, stroke, or transplant require a lifesaving cellular therapy, the best matches are found in similar ethnic and genetic backgrounds.

How can blood banks and the marrow registry provide life-saving therapies (such as blood transfusions and BMT) to Black communities if the safest matches come from similar ethnic groups, but many in these communities aren’t aware of the need, are unfamiliar or untrusting of the process or simply don’t donate as often as they could?!?! Simple…once we know the solution (donate!), we can do something about it!

When we think about blood transfusions for those who have had (or will have) multiple transfusions throughout their lives, it is essential for hospitals to maintain an adequate supply of closely matched blood — which is typically found in donors of the same or similar ethnicity.

Lend a hand by lending an arm!

African American blood donors play a critical role in helping to ensure that patients battling sickle cell disease receive the most compatible blood match. There aren’t enough blood donors who are African American to meet this urgent need. In 2020, the American Red Cross launched a national SCD initiative to ensure the availability of donor blood to individuals needing blood transfusions.

Clinical Trials