December 18, 2024
In her monthly column "Cell Notes," AABB's Christina Celluzzi, PhD, MS, CABP(H), shares insights, findings and commentary on emerging topics in biotherapies. Subscribe to CellSource, AABB's biotherapies newsletter, to receive "Cell Notes" and the latest news directly in your inbox.
As we round out the year, it’s a perfect time to reflect on the remarkable advancements in biotherapies and to look forward to the exciting innovations on the horizon. The holiday season, with its sense of renewal, offers an ideal backdrop to consider these developments and what they promise for the coming year.
The field of biotherapies is poised for significant breakthroughs. Researchers are expanding treatments to target solid tumors like lung, kidney, pancreatic and ovarian cancers. Efforts to enhance safety and efficacy are underway, with CAR natural killer (NK) cell therapies showing promise in clinical trials. The use of autologous therapies, where a patient’s own cells are used, is becoming more prevalent, reducing the risk of immune rejection. Advances in gene editing technologies, such as CRISPR, are enabling more precise modifications of cells to enhance their therapeutic potential. Additionally, there is a concerted effort to make cell therapies more accessible and affordable by streamlining the manufacturing process and reducing costs.
2024 has also seen significant advancements in CRISPR-Cas9 technology, with new variants reducing off-target effects and making gene editing safer and more reliable. CAR-T cell therapy has improved, with next-generation CAR-T cells targeting multiple cancer antigens simultaneously, increasing their effectiveness.
Gene therapy has shown promise for rare genetic disorders, such as spinal muscular atrophy (SMA), leading to significant improvements in patients’ motor function and quality of life. One of the most notable advancements this year in gene therapy is exagamglogene autotemcel (Casgevy, Vertex Pharmaceuticals), a therapy approved for treating sickle cell disease in patients aged 12 years and older with recurrent vaso-occlusive crises (VOC).
Exagamglogene autotemcel uses the patient’s own cells, which are collected and edited using CRISPR/Cas9 technology. These cells then target the BCL11A gene, enabling the production of fetal hemoglobin (HbF), which prevents the sickling of red blood cells. These modified stem cells, when transplanted back into the patient, engraft in the bone marrow to produce healthy cells—an innovative approach that has shown success in clinical trials, with many patients experiencing a substantial reduction in VOCs.
Stem cell therapy has made significant progress in treating neurodegenerative diseases like Parkinson’s and Alzheimer’s. Promising studies on Parkinson’s disease have shown that transplanted stem cells, designed to develop into dopamine-producing neurons, successfully integrate into the brain. These transplanted cells continue to signal dopaminergic activity, producing and releasing dopamine, a crucial neurotransmitter for controlling and coordinating body movements. The hope is that infusing the brain with these specialized cells will restore critical dopamine production and slow the progression of the disease, offering new hope for patients.
Immunotherapy has advanced, particularly in treating solid tumors, with new immune checkpoints and inhibitors boosting the immune system’s ability to target cancer cells. Artificial intelligence (AI) has played a crucial role in accelerating biotherapy research, optimizing treatment plans, and identifying new drug targets.
The progress in biotherapies is a result of the collaborative efforts of scientists, researchers, and healthcare professionals worldwide. Organizations like AABB play a crucial role in advancing these therapies. AABB supports the field through standards setting, accreditation, education, policy efforts, member activities and more.
As we look forward to 2025, we carry with us gratitude for the scientists and health care professionals whose dedication and hard work have brought us these groundbreaking therapies. Their efforts remind us of the importance of perseverance and the impact of collective effort. Let’s carry forward the spirit of hope, collaboration and gratitude into the new year. The advancements in biotherapies are a beacon of what we can achieve together, promising a future where more lives are transformed and saved.
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