The United States has fallen behind other countries in the effort to make pancreatic islet cell transplants available for patients with type 1 diabetes. As the technique grows in popularity through much of the developed world, in America innovation has come to a standstill.
Islet transplantation is an advanced treatment for type 1 diabetes. Briefly, doctors take a donor’s islets of Langerhans (the clusters of cells in the pancreas that contain the insulin-producing beta cells) and inject them into patients with diabetes, typically into the liver. When successful, patients can discontinue insulin use altogether, or greatly reduce it. Some transplant recipients have remained free of insulin therapy for many years following the procedure, with only minor side effects, although most patients require some insulin therapy at five years post-transplant.
Islet cells are harvested from the pancreas of a deceased organ donor, and immunosuppressive drugs are required to prevent rejection by the recipient’s immune system. These donor cells are scarce, and while there are many ongoing attempts to find other sources of usable islet cells (such as the use of pluripotent stem cells or even genetically engineered pigs), the procedure is not expected to become a mainstream treatment in the foreseeable future. But for patients with a dire need—for example, those with extreme glucose management challenges, hypoglycemia unawareness, or advanced kidney disease—islet transplantation can be a lifesaver.
Unfortunately, in the United States, the procedure has been “effectively halted,” according to an official report on the American Diabetes Association’s recent Scientific Sessions conference. While the technique was largely investigated and defined by scientists working in the US, it has never become available to the general public, and there is little hope that it will become available any time soon. Meanwhile, some medical centers in Canada, Australia, Europe, and Asia are now performing the technique routinely.
Dr. Camillio Ricordi, a leading researcher on the treatment with the University of Miami, blamed the issue on “outdated regulation” from the FDA. Because of a quirk in the National Organ Transplant Act of 1983, subparts of organs (such as the islets of Langerhans) don’t count as organs and are not covered by the network of rules and programs governing organ transplantation. Instead, transplanted islet cells are regulated as if they were drugs. Treating the cells as pharmaceuticals subjects them to standards for precision and consistency that they cannot meet.
Phase 3 trials have already proved that islet cell transplantation can deliver impressive results, and do so safely. And yet, America’s leading experts have agreed that it is all but impossible for a research institution to apply for and secure full approval for the technique.
Around the world, other medical authorities have taken a more practical stance. This has also allowed insurers to pay for the procedure, just as they would any other organ transplant. Most of the countries that lead the world in islet transplantations have robust social healthcare systems.
But islet transplantation is still considered purely experimental in the United States, and today can only be performed under the auspices of an official clinical trial. While the ADA and other advocates have pushed for change, so far, the regulation still stands in the way.
As islet transplantation becomes more available across the globe, the treatment is going nowhere fast in the USA.