Fifty years ago, the idea of altering a gene to treat or even cure a disease was considered science fiction. Today, cell and gene therapy are transforming treatment options for some patients and are part of an exciting new era of medicine.
A new report released today finds there are 289 novel cell and gene therapies in development for a variety of diseases and conditions. The therapies represent the translation of basic scientific insights into innovative new treatment options for patients. These medicines in development are either in clinical trials or awaiting review by the U.S. Food and Drug Administration.
The novel cell and gene therapies in the development pipeline are the result of years of pioneering research. The range of diseases these therapies can address is broad and covers blood and eye disorders, cancer and infectious disease to name a few.
This new field of cell and gene therapy, a major part of the broader field of regenerative medicine, is already making an impact. American Society for Blood and Marrow Transplantation president John F. DiPersio, M.D., Ph.D. noted, “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.”
Justin and Boris, a patient and a biopharmaceutical researcher respectively, know firsthand the impact these treatments can have on individual lives. At the age of seven, Justin was diagnosed with acute lymphoblastic leukemia, a type of blood cancer. He spent more than half his life battling this disease, but a new CAR-T cell therapy finally put him in remission and gave him hope.
Boris is particularly interested in advancing the potential of personalized medicine and CAR-T therapy – the same therapy that saved Justin’s life. “It’s being manufactured or generated for every single patient. We teach [white blood cells] how to fight cancer, and give them back to the patient,” he said. While implementation may be complex, researchers like Boris demonstrate the future for cancer patients has never been more promising.
To learn more about the new advancements in cell and gene therapy, read the full report here.
Andrew Powaleny is Senior Director of Public Affairs at PhRMA and leads the organization's scientific communications. Before joining PhRMA in 2015, he worked in public affairs for a small firm in Washington, DC and served as Deputy Press Secretary for the House Committee on Energy and Commerce. Andrew came to Washington, D.C. via Connecticut with a degree from Eastern Connecticut State University where he majored in public policy and government. Andrew is active as a runner and volunteer with the DC Front Runners; most recently serving on its Board of Directors for three years as co-race director. He is also a member of the NLGJA: The Association of LGBTQ Journalists and mentors students through his alumni association with The Fund for American Studies. Andrew is passionate about scientific innovation, especially for mental illness, and his heroes are the men and women of America’s biopharmaceutical research companies.