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Report – 289 Cell and Gene Therapies in Development

Human T-cell

Scanning electron micrograph of a healthy human T-cell (NIH.gov)

6 Dec. 2018. A report by a pharmaceutical industry organization says nearly 300 new therapies that transfer live cells or genes as treatments for disease are in clinical trials or being reviewed by Food and Drug Administration. The report was issued by today Pharmaceutical Research and Manufacturers of America, or PhRMA, in Washington, D.C.

PhRMA defines cell therapies as the introduction of new cells into patients to grow into replacement cells, or to repair damaged cells or tissue from a disease. In this group, PhRMA includes stem cell therapies, as well as immune system cells such as lymphocytes and dendritic cells, and islet cells in the pancreas that produce insulin. Cell therapies can be derived from donated cells or autologous cells, those from the patients themselves. Among these treatments are synthetic T-cells with chimeric antigen receptors, also called CAR T-cells, new therapies being developed for several types of blood-related and solid tumor cancer.

Gene therapies, says PhRMA, introduce healthy or modified genes into a patient’s cells to produce properly functioning proteins or block damaging proteins from being produced. Included in gene therapies are treatments that edit a patient’s genes to remove mutations causing a disease. with Crispr — clustered regularly interspaced short palindromic repeats — among the more well-known technologies. This group also has gene silencing treatments, such as RNA-interference that harnesses a natural process for turning off production of disease-causing proteins.

Gene transfers can take place in the lab, then infused in the patient, or given transferred directly into the patient, often using benign viruses for delivery. Treatments that genetically alter cells as treatments, such as in CAR T-cells, are a hybrid form of cell and gene therapy.

PhRMA’s review, from government and industry sources as well as the Adis Insight drug development database, shows by a wide margin more cell and gene therapies are being developed to treat cancer than other types of disease. Of the 289 cell and gene therapies in development, 111 or 38 percent are designed to treat some form of cancer. Between 20 and 30 of these therapies are developed as treatments for eye disorders (28), cardiovascular disease (26), neurological disorders (22), and blood disorders (21). All of the other 15 disease categories each have less than 20 new treatments.

While all of the disease categories included cell and gene therapies in various stages of clinical trials, only 2 of the disease groups — neurological disorders and infectious diseases — have treatments currently in FDA review. Cancer, which accounts for nearly 4 in 10 of all new cell and gene therapies, has none of these treatments yet reaching FDA.

PhRMA considers cell and gene therapies part of the broader field of regenerative medicine. A blog post announcing the report on the PhRMA web site quotes John DiPersio, president of American Society for Blood and Marrow Transplantation, who notes, “The field of regenerative medicine is enhancing science, education and clinical care that will provide patients with expanded and genetically modified stem and immune effector cells for the treatment of inherited diseases, solid tumors, and hematologic malignancies to live longer lives.” DiPersio is an oncologist at Washington University in St. Louis medical school.

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