FDA Inaction Endangers Organ Transplant Patients
The Senate will soon vote on President Biden's nominee for FDA Commissioner, Dr. Robert Califf. If confirmed, he should commit to action on a lifesaving new technology that, if no action is taken, could soon gather dust on the shelves.
More than 100,000 Americans are waiting for an organ transplant. Due to a persistent shortage of organs, many of these patients have been languishing in pain for years. Surgeons performed only 39,000 transplants in 2020 because of this shortage.
It doesn't have to be that way. Astounding new technology known as 3D bioprinting has the potential to create artificial organs. That could narrow the shortage significantly -- and save or extend roughly 900,000 lives per year, given the prevalence of organ failure or compromise in the health of so many people.
3D bioprinting involves harvesting a patient's own cells, letting them replicate and multiply outside of the body, and then using them to "print" living tissue for transplantation.
Unfortunately, the Food and Drug Administration, which has jurisdiction over breakthrough treatments like this, has not provided the guidance necessary to evaluate the safety and efficacy of 3D bioprinting. Unless the FDA establishes the proper regulatory framework for evaluating and approving 3D bioprinted organs, thousands of patients could die unnecessarily while awaiting a transplant.
3D bioprinting has shown amazing results for patients with desperate medical conditions. In 2006, Dr. Anthony Atala and his team at Wake Forest University's Institute for Regenerative Medicine successfully implanted the world's first lab-grown bladders into seven children born with spina bifida. This neural tube defect can cause bladder malfunctions that lead to kidney failure. Patients may face a lifetime on dialysis.
The Wake Forest team is now in the process of researching bioprinting for nearly 40 tissues and organs. Its research has led to the creation of "kidney units" generated from patient tissue samples. They’re now moving to phase 3 clinical trials for patients with end-stage renal failure. About 80% of all transplant patients are waiting for kidneys. If the Wake Forest team is successful, their efforts could save thousands of lives.
Similarly, Techshot, an Indiana-based aerospace company, has also seen promise in micro-gravitational 3D bioprinting. In 2019, Techshot established the BioFabrication Facility on the International Space Station. The project has already printed cardiac cells in space and aims to successfully print transplantable cardiac tissue within the next 10 years.
Yet there's no regulatory framework for speedily approving technology like this, as a new documentary, "They Say It Can’t Be Done," points out. Several of Dr. Atala's patients have benefited from his breakthrough technology.
Back in 2017, the FDA issued a statement promising to "review the regulatory issues related to the bioprinting of biological, cellular and tissue-based products in order to determine whether additional guidance is needed." That's the last that has been heard from the agency on the subject.
Inaction is hampering the rollout of bioprinted organs. The industry is still in its infancy, and startups depend on large amounts of venture capital to complete their research. If investors aren't sure how long it will take for the FDA to evaluate new technologies -- or even what sort of approach regulators would adopt -- they will be less likely to put up the funding for this lifesaving research.
It turns out Americans in need of an organ transplant now go on two waiting lists. One is for the critical organ. The other is for the FDA to inform us how to assess and approve new life-saving technologies. The agency's new leadership should commit to ending this wait.
Dan Troy is the former chief counsel for the FDA, where he served as a primary liaison to the White House and the US Department of Health and Human Services.
