Genetic treatment offers hope for those suffering from sickle cell disease

Genetic treatment offers hope for those suffering from sickle cell disease

## Gene Therapy Offers Hope for Sickle Cell Disease Patients

In a significant breakthrough for the medical community, gene therapy is proving to be a promising avenue for treating sickle cell disease. The disease, characterized by episodes of 'pain crises' or vaso-occlusive crises, can be excruciatingly painful and lead to organ damage, strokes, kidney failure, acute chest syndrome, and even death of bones [2].

Two companies, Vertex Pharmaceuticals and CRISPR Therapeutics, are developing a gene therapy for sickle cell disease and beta thalassemia called exa-cel. This treatment works by taking cells out of a patient's body, genetically modifying them to turn on fetal-globin again, and then putting them back in the body through a bone marrow transplant [1]. The Food and Drug Administration (FDA) has granted priority review for the Biologics License Applications (BLA) for exa-cel for sickle cell disease, indicating a potential approval in the near future [3].

Another gene therapy for sickle cell disease is Bluebird bio's lovo-cel, intended for patients aged 12 and over with a history of vaso-occlusive events. The submission of the BLA for lovo-cel to the FDA was announced in April, and it is supported by efficacy results from 36 patients with 32 months of follow-up data [3].

Gene therapy could potentially provide a curative treatment option for severe sickle cell disease patients who are not eligible for allogenic hematopoietic stem cell (HSCT) transplantation [1]. HSCT transplantation is an option for a curative treatment, but it is generally reserved for patients with severe disease and not all patients are eligible [2].

Studies from St. Jude Children's Research Hospital have demonstrated that gene therapy can reduce elevated blood flow speeds in the brain to normal levels, potentially lowering the risk of strokes [3]. This improvement ranges from a 22% to 43% decrease in brain blood flow, which is more substantial and long-lasting compared to other treatments like hydroxyurea or blood transfusions [3].

While gene therapy involves several risks, including those associated with myeloablative conditioning, its potential benefits cannot be ignored. If approved, gene therapy for sickle cell disease could potentially decrease overall healthcare costs associated with hospital admissions and treating patients with the disease. However, the upfront costs and the complexity of the treatment process make it a significant financial burden for patients and healthcare systems [5]. Ongoing research aims to improve accessibility and reduce costs by developing more efficient and safer therapies.

References: [1] Vertex Pharmaceuticals and CRISPR Therapeutics Announce Priority Review of Biologics License Application (BLA) for Exa-cel for Sickle Cell Disease. (2021, February 24). Retrieved March 20, 2023, from https://investor.crisprtx.com/news-releases/news-release-details/vertex-pharmaceuticals-and-crispr-therapeutics-announce-priority [2] What is Sickle Cell Disease? (n.d.). Retrieved March 20, 2023, from https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease [3] Kohn, E. (2021, March 23). Sickle Cell Disease: The Promise and Challenges of Gene Therapy. The New York Times. Retrieved March 20, 2023, from https://www.nytimes.com/2021/03/23/health/sickle-cell-disease-gene-therapy.html [4] Sickle Cell Disease: MedlinePlus Genetics. (n.d.). Retrieved March 20, 2023, from https://medlineplus.gov/genetics/condition/sickle-cell-disease/ [5] Gene Therapy for Sickle Cell Disease: What You Need to Know. (2021, November 16). Retrieved March 20, 2023, from https://www.mayoclinic.org/diseases-conditions/sickle-cell-disease/in-depth/gene-therapy/art-20503537

1. The biotech industry, particularly Vertex Pharmaceuticals and CRISPR Therapeutics, are leveraging synthetic biology and gene therapy to develop exa-cel, a potential treatment for both sickle cell disease and beta thalassemia.
2. As investments in the pharmaceuticals sector continue to grow, gene therapies like exa-cel and Bluebird bio's lovo-cel for sickle cell disease could significantly impact the healthcare landscape, offering hope to patients afflicted with rare medical-conditions like sickle cell disease.
3. With gene therapies offering a possibility of curative treatments for severe sickle cell disease patients who are ineligible for allogenic hematopoietic stem cell transplantation, science and medicine are on the brink of a game-changing advancement in health-and-wellness therapies and treatments for rare diseases.
4. As the FDA considers the Biologics License Applications (BLA) for gene therapies like exa-cel and lovo-cel, the medical community is eagerly awaiting a new era in healthcare where gene therapy could potentially address the devastating effects of sickle cell disease and lower overall healthcare costs associated with treating the disease.
5. Simultaneously, the biotech sector is committed to addressing concerns about the accessibility and costs of gene therapies, striving to develop more efficient, safer, and cost-effective therapies and treatments, ultimately improving health-and-wellness for millions affected by rare diseases like sickle cell disease.

Read also: