FDA Raids One Stem Cell Lab and Warns Another in Regulatory PushBy
Agency to get tough on those touting unproven cell therapies
Regulator to help legitimate developers gain sales approval
U.S. health authorities will more closely regulate a burgeoning, and sometimes unproven, field of medicine that employs the body’s own cells or tissues to help treat a wide variety of diseases or conditions.
On Monday, the Food and Drug Administration released a warning letter it sent last week to Florida-based U.S. Stem Cell Inc. for selling unapproved stem-cell treatments and injecting them into patients’ spinal cords. And it raided a San Diego-based stem cell treatment center and seized vials of a live virus typically reserved for people at high risk of smallpox that was being mixed with stem cells and injected directly into cancer patients’ tumors.
More enforcement will be coming, FDA Commissioner Scott Gottlieb said Monday. The agency also plans to create a working group to pursue unscrupulous clinics.
“The prospect of clinical tissue repair strategies is a tangible reality,” Gottlieb said in a statement. However, some businesses have seized on that promise and made “deceptive, and sometimes corrupt, assurances to patients based on unproven and, in some cases, dangerously dubious products.”
Known as regenerative medicine, the practice uses human cells or tissues that are engineered or taken from donors. The market stretches from small companies developing treatments to individual clinics where doctors offer procedures often involving patients’ own stem cells or tissues.
Over the next several months, the FDA plans to clarify which treatments will fall under its oversight, and tell providers of the therapies what they need to do to get regulators’ approval.
For example, some individualized treatments where doctors at a clinic remove a patient’s stem cells and put them back in with minimal manipulation likely won’t be subject to FDA review, Gottlieb said. However, when the cells are manufactured on a larger scale, the FDA would police the product, he said.
The effort to boost legitimate regenerative therapies is part of broader legislation known as the 21st Century Cures Act, which was signed by then-president Barack Obama last year. The law directed the FDA to provide extra guidance to companies making regenerative therapies for serious or life-threatening illnesses, or that filled an unmet medical need.
The agency has issued some direction for companies seeking to sell regenerative therapies, but that will be reworked as part of guidance in the coming months.
Vericel Corp. is one of the companies that’s already been designated to receive the additional agency help for its experimental treatment for some patients with advanced heart failure. The therapy is manufactured from a patient’s own bone marrow, with the goal of reducing inflammation and getting blood vessels to grow and help repair damaged tissue.
Like Vericel, with a market valuation of about $113 million, many of the companies in the field are small. The FDA said it plans to make it easier for the companies, which typically don’t have the large regulatory departments of giant drugmakers, to meet the agency’s demands.
“Ultimately, the agency’s goal is to make sure that the potential of regenerative medicine can continue to advance to benefit the patients who need new and innovative options for their medical problems,” Gottlieb added. “These technologies hold out the potential to significantly alter the course of a broad range of diseases.”