The American population is getting older and increasingly suffering from degenerative, chronic conditions. Between 2010 and 2050, the United States population ages 65 and older is expected to double from about 40 million to 84 million people. A significant majority of older Americans suffer from at least one—and often many—chronic conditions, which is expected to drive additional costs and place growing pressure on the health care system. These trends call for new, innovative strategies to keep people healthy.
The primary tools of twentieth century medicine—drugs, medical devices, and surgical interventions—have proved to be remarkably successful in eliminating disease and extending lifespan. But success has been limited with respect to many congenital, age-related, and trauma-induced injuries and diseases involving organ and tissue degeneration. The reigning clinical paradigm emphasizes treatment—palliation and symptom control—rather than curative therapy aimed at resolving the root cause of disease. Furthermore, in most cases, these traditional therapies have a deleterious effect on normal tissues and organs, often resulting in side effects and long-term dysfunction. There is a promising new approach to address degenerative organ and tissue disease and damage: the ability to use human cells as a viable, therapeutic option to rejuvenate, regenerate, or replace diseased organs and tissue.
These treatments will not only help patients, they also have the potential to create savings in the health care system by replacing high-cost surgeries and drugs with less expensive, outpatient procedures. For example, if diabetes were to be cured through the permanent or semi-permanent replacement of insulin-producing cells, then the lifetime cost and invasiveness of daily insulin injections would be eliminated.
Despite the promise of these new treatments, the existing statutory framework of the Federal Food, Drug, and Cosmetic Act and the Public Health Service Act do not address cell therapy directly. Today’s regulations largely prevent a patient from using his or her own cells to treat many medical conditions: a problem that this report seeks to address.
This report offers recommendations to accelerate the availability of safe and effective cellular therapies to Americans in need and improve U.S. competitiveness in the global marketplace. The report builds on initial policy recommendations included in the BPC report, Advancing Medical Innovation for a Healthier America, as well as a technical assistance letter provided to the chairman and ranking member of the Senate Health, Education, Labor, and Pensions (HELP) Committee, both released in July 2015.
Overview of Cellular Therapy
Regenerative medicine is an emerging field that seeks to restore health rather than merely treat disease. As the National Institutes of Health explains, “Regenerative medicine is the process of creating living, functional tissues to repair or replace tissue or organ function lost due to age, disease, damage, or congenital defects. This field holds the promise of regenerating damaged tissues and organs in the body by stimulating previously damaged tissue and irreparable organs to heal themselves.” Central to the practice of regenerative medicine is cellular therapy, or the use of therapeutic cells to restore healthy organ and tissue function.
Every living tissue in the human body is comprised of cells and they are responsible for carrying out the function and maintenance of every organ in the body. Cells and natural combinations of cells have been used safely and successfully for therapeutic purposes for more than fifty years. Blood transfusions were the first type of cellular therapy and bone marrow transplantation has been a standard of care for patients with aggressive forms of cancer for decades. Organ transplants have become routine in modern medicine and have saved countless lives, while grafts of the skin and cornea for burns or eye injury have been widely employed.
But recent scientific progress using cells derived from either perinatal or adult tissue has significantly advanced the capacity to use cellular therapies across many other medical specialties, including cardiology, neurology, ophthalmology, orthopedics, organ transplantation, urology, and others.
By providing healthy, functional tissues and organs, regenerative medicine will improve the quality of life for individuals. The National Institutes of Health describes the long-term promise of regenerative medicine as a world where there is no donor organ shortage, where victims of spinal cord injuries can walk, and where weakened hearts are replaced.