{"id":6854,"date":"2023-12-08T23:18:20","date_gmt":"2023-12-08T23:18:20","guid":{"rendered":"https:\/\/www.sicklecelldisease.org\/?p=6854"},"modified":"2023-12-14T21:59:11","modified_gmt":"2023-12-14T21:59:11","slug":"scdaa-statement-about-gene-therapy-approval","status":"publish","type":"post","link":"https:\/\/www.sicklecelldisease.org\/2023\/12\/08\/scdaa-statement-about-gene-therapy-approval\/","title":{"rendered":"SCDAA Statement About Gene Therapy Approval"},"content":{"rendered":"

On Dec. 8, 2023, the Food and Drug Administration (FDA) approved two cell-based gene therapies for sickle cell disease (SCD), Casgevy from CRISPR\/Vertex and Lyfgenia from bluebird bio. These are the first treatments of their kind available to individuals with SCD in the United States. SCDAA welcomes the approval of these potentially curative therapies which mark major advances in the treatment of sickle cell disease; however, there are valid concerns about accessibility and the potential for adverse effects.<\/p>\n

Dr. Lewis Hsu, chief medical officer of the Sickle Cell Disease Association of America Inc. said:<\/p>\n

We at the Sickle Cell Disease Association of America Inc. celebrate that two gene therapies are approved for sickle cell disease by the FDA today, Dec. 8. This double milestone was a long time coming, and sickle cell disease now joins the ranks of other genetic diseases with gene therapy treatments. Sickle cell disease was called \u201cthe first molecular disease\u201d about 70 years ago, and a gene therapy treatment was predicted for sickle cell disease in the 1950s when DNA was first described.<\/p>\n

These two gene therapies mark a big step forward for sickle cell disease research and treatment. The results of the clinical trials are very impressive. The patients selected had a lot of pain (two or more vaso-occlusive crisis pain hospitalizations per year for two years). The data show that, after gene therapy was administered, nearly all patients were free of hospitalizations for vaso-occlusive crises for at least nine consecutive months. The patients\u2019 health-related quality of life also improved in every way: physically, emotionally, socially and functionally with both gene therapies.<\/p><\/blockquote>\n

Regina Hartfield, president and CEO of the Sickle Cell Disease Association of America Inc. said:<\/p>\n

Gene therapy is an exciting and potentially curative addition to the treatments available to sickle
\ncell warriors. This is a historic milestone, but everyone may not be eligible for gene therapy.
\nWe must continue to move forward with research to ensure that there is a solution for every member of our community.<\/p><\/blockquote>\n

Is gene therapy a cure for sickle cell disease?<\/strong>
\nThe Sickle Cell Disease Association of America Inc. recognizes gene therapy as a \u201cpotentially curative\u201d therapy. The treatment is so new that more data is needed to understand its impact and patient prognosis. Additionally, the word \u201ccure\u201d suggests a simple solution that does not reflect the reality of these therapies. Even after completing treatment, the FDA recommends 15 years of patient monitoring for health issues.<\/p>\n

What is the treatment like?<\/strong>
\nThe patient journey will be similar for both Casgevy and Lyfgenia. Gene therapy is administered during a one-time infusion; however, there are steps that patients must take to prepare for the treatment. First, the patient\u2019s care team will collect stem cells, which are the progenitors of red blood cells, from their body. Then, those cells will be treated in a lab. The patient will undergo chemotherapy to remove the original, abnormal stem cells from the bone marrow. After this process is complete, the treated stem cells are injected back into the patient through an intravenous process like a transfusion (not surgery). The whole procedure takes about a year. It is similar to autologous bone marrow transplantation, as there is no need to find a donor of stem cells.<\/p>\n

What is the difference between the two therapies?<\/strong>
\nThe two gene therapy strategies are scientifically different. Casgevy is gene editing, the first of its kind, and Lyfgenia uses gene addition. Both gene therapy strategies have about the same patient journey and potential issues: access, cost, infertility, unknown possibility of organ damage and unknown long-term effects.<\/p>\n

How effective is it?<\/strong>
\nGene therapy provides a significant reduction in acute episodes of sickle cell pain within a few years of administration. More years of follow-up will be needed to determine whether it will also reduce the organ damage of sickle cell disease and if the stem cells treated with continue to produce non-sickling red blood cells for the rest of the person\u2019s life, or if the stem cells die off over a certain number of years. Currently the treatment requires chemotherapy, which means there are also concerns about chemotherapy-associated complications, such as infertility or secondary cancer.<\/p>\n

When will gene therapy be available for use?<\/strong>
\nGene therapy will likely be available in early 2024.<\/p>\n

Who is eligible?<\/strong>
\nCasgevy and Lyfgenia are approved for people ages 12 and up. Sickle cell disease SS and S-beta-zero-thalassemia are eligible. The FDA indicates that sickle cell disease SC is not included. Additionally, individuals may also not be able to receive gene therapy if they have:<\/p>\n