Gene Therapy Treatments for Sickle Cell Patients Maybe Inaccessible for Many
Is Gene Therapy Really for All?
History of Sickle Cell Disease Treatment
Historically, Sickle Cell Disease has been treated primarily with blood transfusions and medications such as Hydroxyurea. However, neither of these methods can cure the disease. Blood transfusions can help for a short while, but in order to maintain the benefits, they must be performed regularly, or the effects wear off. As for hydroxyurea, the medication can increase the amount of effective hemoglobin in a person’s body which can reduce the frequency of pain crises a person experiences. However, the medication does not completely prevent crises from occurring.
In 1984, an eight-year-old girl with Sickle Cell Disease was given a bone marrow transplant after developing leukemia. After the transplant, the young girl was shown to not only recover from leukemia, but also from Sickle Cell Disease. While her body was still producing some of the problematic hemoglobin, the bone marrow transplant had given her the ability to also produce healthy hemoglobin, enough to stop her from experiencing any further pain crises. This was the first case of a bone marrow transplant being used to treat Sickle Cell Disease.
New Methods Becoming Available
Thirty-five years later, bone marrow transplants are still performed to attempt to cure Sickle Cell Disease. However, the procedure is not without problems. Many people who undergo the procedure develop what is called “graft-versus-host disease” where the transplanted cells attack the patient’s other cells. This occurrence is extremely severe and can cause multiple issues, sometimes even death. In addition to this risk, bone marrow transplants can only be performed when a “match” is found between the patient and a potential donor. Currently, only about one third of patients are matched to a donor.
Currently, only about one third of patients are matched to a donor.
Recently, a new method of treatment has begun to be tested, known as gene therapy. This method works similarly to bone marrow transplantation in that it helps the patient’s body to create healthy hemoglobin. However, instead of needing a matched donor, the patient’s own stem cells are modified to be able to make the healthy hemoglobin by themselves. This not only eliminates the risk of graft-versus-host disease, but it makes the procedure accessible to mostly anyone, not just those who have a matched donor.
The gene therapy method is currently undergoing clinical trials and shows great potential as a future method and possible cure for Sickle Cell Disease in some patients. However, there are some drawbacks to the method as well.
Is it “Universally” Accessible?
Perhaps the biggest advantage of gene therapy is that it can be done in mostly any patient, not just those who match to a donor. But, is it really universally accessible? Many patients already struggle to gain access to bone marrow transplant services due to the huge cost of such care. A bone marrow transplant typically garners a price tag of up to $250,000 or more, so even if a patient does find a matched donor, paying for the treatment and getting approved can be immense hurdles to overcome.
The price tag on gene therapy has not yet been set, mostly due to its so far not being approved by the FDA as a treatment. However, preliminary numbers and some research articles are suggesting that the procedure will cost as much as $1,000,000. This number is astronomical, and for a “universal” cure, it certainly prevents a large number of patients from accessing its benefits.
Current estimates show that over half of Sickle Cell Disease patients in the United States do not have private insurance, instead relying on Medicare or Medicaid to receive healthcare. Even for those that do have private insurance, these companies often set limits on how much they will contribute to procedures such as these, leaving the patient to still pay a hefty portion of the cost out-of-pocket.
Current estimates show that over half of Sickle Cell Disease patients in the United States do not have private insurance, instead relying on Medicare or Medicaid to receive healthcare.
Currently, there is a program in place with the National Institutes of Health (NIH) by which they fund for Sickle Cell Disease patients to receive bone marrow transplant or gene therapy treatment. However, this program is extremely limited; the spots available for funding fills up very quickly and the criteria to qualify for such funding is extremely narrow. While this option does allow for some patients to access these costly treatments, it is not a realistic program for aiding large numbers of people.
Hope for the Future
Gene therapy is certainly an excellent move in the right direction when it comes to curing Sickle Cell Disease in some patients. It is a treatment that hopefully ends Sickle Cell complications and symptoms without the need for repeated use, and it is a treatment that can be done for qualifying patients. However, the current forecast on pricing for this procedure suggests that it may not be the miracle cure that patients have been looking for. Moving forward, it is important for Sickle Cell patients and advocates to speak up about this treatment option and find ways of making it accessible to everyone, regardless of insurance, health limitations or financial status. We are still in search of a universal cure, so our fight will not end.
Disclaimer: All information contained in the Sickle Cell 360 blog is intended for general, eduational or background purposes only and should not be used as medical advice for any individual. Please consult your health-care provider or providers for information specific to your own condition or illness. If you need assistance locating a medical doctor or other health-care provider, please call us at 1-844-994-Hope or email us at firstname.lastname@example.org.
Fitzhugh, C. D., Abraham, A. A., Tisdale, J. F., & Hsieh, M. M. (2014). Hematopoietic stem cell transplantation for patients with sickle cell disease: progress and future directions. Hematology/Oncology Clinics of North America, 28(6), 1171-1185. doi: 10.1016/j.hoc.2014.08.014
Lothe, P., Pompa, T., Jain, M., Patel, P., Liang, Y., Yalamanchi, A., Ward, K., & Styler, M. (2015). Comparative cost analysis of therapy in sickle cell anemia: supportive care vs. bone marrow transplant. Blood, 126(23), 4466.
National Institutes of Health. (2018). NIH launches initiative to accelerate genetic therapies to cure sickle cell disease. https://www.nih.gov/news-events/news-releases/nih-launches-initiative-accelerate-genetic-therapies-cure-sickle-cell-disease
Nowogrodzki, A. (2018). Gene therapy targets sickle-cell disease. Nature Outlook: Gene therapy, 564, S12-S13. doi: 10.1038/d41586-018-07646-w