Therapeutic innovation drives patient treatment updates and improves patient outcomes. From the development of chemotherapy to monoclonal antibody use in cancer treatment, the ability to target aggressive disease is quickly advancing. Emerging therapeutics can improve the prognosis of conditions related to neurodegenerative and cardiovascular diseases, as well as cancers, reducing the burden of such diseases. This blog post will outline major therapeutic breakthroughs that will likely shape the medical landscape in the upcoming year.
CRISPR-Cas9
This December, the FDA approved both Casgevy and Lyfgenia. These cell-based therapies are used to treat Sickle Cell Disease (SCD), a blood disorder affecting approximately 100,000 Americans. Casgevy is the first FDA approved drug to use clustered regularly interspaced short palindromic repeats or CRISPR and Cas9 technology. CRISPR is a technology utilized by researchers for DNA modification, and Cas9 is an enzyme used by bacteria to cut DNA. Together, these mechanisms can be used to edit genomes and correct mutations. Casgevy leverages this technology to help the body produce fetal hemoglobin and reduce symptoms associated with SCD. Beyond SCD, CRISPR demonstrates potential applications in enhancing cancer treatment, slowing the progression of familial transthyretin amyloidosis, and reducing populations of malaria-infected mosquitoes.
BTK Inhibitors
B-cells in humans play a key role in immune responses by producing antibodies, however uncontrolled B cells can lead to lymphomas and leukemias. Inhibiting bruton’s tyrosine kinase (BTK), which is the precursor to the development of B cells, is revolutionary, as BTK inhibitors can stop the uncontrollable replication of B cells leading to treatment of certain cancers. The first BTK inhibitor was discovered in 1999, though clinical trials for novel drugs using this mechanism have taken place as recently as 2021. Currently, there are four FDA approved BTK Inhibitors, Ibrutinib, acalabrutinib, zanubrutinib, and pirtobrutinib, all of which are used to treat mantle cell lymphoma. New research suggests that BTK inhibitors may also be useful for the treatment of inflammatory and autoimmune conditions by reducing inflammatory immune responses in the body.
CAR-T Cell Therapy
T-cells are another important lymphocyte involved in the immune response that destroy cells within the body that have been attacked by a virus or cancer. CAR-T cell therapy implements gene therapy to efficiently use T-cells to get rid of cancer-causing B cells or other antigens. A patient’s T-cells are separated from their blood and engineered to produce chimeric antigen receptors or CAR. Adding the genetic material for CAR helps T-cells attach to specific cancer-causing antigens. This type of therapy is typically used for cancer, though recent research highlights the potential for CAR-T cell therapy to treat autoimmune disorders. Autoimmune diseases cause the body’s immune system to attack its own healthy cells, which can lead to debilitating symptoms. Antibodies, produced by B-cells, play a significant role in autoimmune conditions’ overactive immune responses. CAR-T cell therapy has only recently been used to target B cells in patients with certain autoimmune conditions as a method to reduce symptoms. The treatment has been successful in patients with autoimmune diseases such as systemic sclerosis, idiopathic inflammatory myositis, and myasthenia gravis. These discoveries open the door for future autoimmune disease treatment options.
The rapidly evolving field of therapeutic innovation is reshaping the landscape of medical treatment, promising brighter prospects for patients across various health conditions. From the groundbreaking applications of CRISPR-Cas9 technology to revolutionary BTK inhibitors offering new avenues in cancer treatment and autoimmune disorders, these advancements underscore the remarkable progress in personalized medicine. Therapeutic interventions not only enhance patient outcomes but also alleviate the burden of debilitating diseases, marking a significant stride towards a healthier and more resilient society.
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