One cannot dispute the advancement in technology when it comes to sickle cell. Decades ago, the management of sickle cell used to be a traditional pain treatment. This used to include over-the-counter medicine, heating pads and increasing fluid intake. Dehydration complicates the disease and makes the symptoms worse, we were told back then, and this is true till today. So, drinking plenty of water in order for the blood cells to move around the body without getting stuck together is vital. The most common pain medications include Ibuprofen, naproxen, diclofenac and nonsteroidal anti-inflammatory drugs (NSAIDs). In more severe cases, narcotics such as meperidine, morphine, and others. Narcotic abuse and addiction are issues that must be considered in any pain control plan. In more severe cases, this will involve going to the hospital to be treated.
As we know, sickle cell disease (SCD) changes the shape of red blood cells. In order to understand gene therapy, one needs to recognise that sickle cell disease is caused by a mutation in the beta-globin gene. This mutation causes blood-forming stem cells, which can self-renew and make all types of blood and immune cells, to produce rigid, sickle-shaped red blood cells. These sickle-shaped cells die early, leaving an insufficient number of red blood cells to carry oxygen throughout the body. Normal red blood cells live in our bodies for 120 days but their sickle-shaped counterparts live for anything between 10 and 20 days. Because of their sickle shape, these cells also get stuck in blood vessels, blocking blood flow, and resulting in excruciating pain. SCD can trigger renal complications, asthma, kidney damage, stroke, and other conditions. Promising new treatments are now available and one of such is called genetic therapies. These are being studied to make sure that they are safe and effective. Genetic therapies can offer a potential cure for SCD.
In recent years, gene therapy has emerged as a promising treatment for SCD. The cells used in genetic therapies, called stem cells, come from a patient’s bone marrow. Bone marrow is the soft, spongy area in the larger bones of the body that creates all cell types that make up the blood. Stem cells are stimulated to exit the bone marrow and enter the bloodstream for collection using a procedure called apheresis. Then, they are modified in the lab and given back to the patient. These stem cells can now make healthy, round red blood cells in addition to other blood cells. For genetic therapies to work, a patient must go through a procedure called conditioning.
What is conditioning? Conditioning is the process of getting a patient ready for genetic therapies. This is done by using a chemotherapy drug. There are two types of conditioning: myeloablative and reduced intensity. Myeloablative conditioning often uses a drug, Busulfan. Reduced-intensity conditioning typically uses another drug, Melphalan.
When and where does conditioning happen? Conditioning happens after the stem cells have been modified in the lab. It allows the stem cells to be returned to the patient. Conditioning is done in a hospital and the patient could have to stay for several weeks after conditioning begins.
Why is conditioning needed and how does conditioning work? A patient’s bone marrow needs to have space for the new stem cells created by genetic therapies. This lets them grow and makes new blood cells. Without this space, the modified stem cells will not survive, and the treatment will not work.
How do I prepare for conditioning?
Life Planning
Life planning should be a part of preparing for conditioning. This includes making plans about your family, home, finances, pets, and employment. You should also think about completing a legal document called an “advanced directive” (including a living will, durable power of attorney, and healthcare proxy). A lawyer or social worker should be able to help with these documents.
Medical Procedures Making sure that you are mentally and physically ready for the procedure is essential. A physician will run tests to be sure that you can have the procedure. In addition to the chemotherapy, you may have to take other drugs before, during and after the procedure. These drugs can help lessen the side effects of conditioning. To minimise the number of intravenous lines and needle sticks, a thin flexible tube called a central line, will be placed into a large vein in your upper chest. It is important to keep the area clean and watch for signs of infection. You should ask your doctor how to properly care for the central line. While patients stay in hospitals for several weeks after conditioning (because the risk of infection is a lot higher during that period), your health will be watched closely. You will also receive check-ups for several years after conditioning.
What side effects can happen with conditioning? There are a number of side effects that can happen with conditioning. Some things that can happen quickly are hair loss, rash, nausea or vomiting, and infections, some of which can be life-threatening. Things that can take longer to show up are tooth and mouth issues, not being able to have children, cancers, or liver and bone damage. A full list of risks should be given to you before starting conditioning. Talk with your doctor to help figure out the most likely risks.
www.curesickle.org
https://stemcell.ucla.edu/news/ucla-researcher-receives-nih-grant-develop-more-affordable-gene-therapy-sickle-cell-disease
If you would like to get in touch with me about sickle cell, do so via email: [email protected] And do check out my blog: https://www.dailylivingwithsicklecell.com/ My book on Sickle Cell – HOW TO LIVE WITH SICKLE CELL and my other books are available for purchase on www.amazon.com
