Regulatory crackdown on unproven stem cell treatments

Associate Professor Megan Munsie, deputy director of the Centre for Stem Cell Systems, joins academics pushing for tighter regulation from the Therapeutic Goods Administration (TGA).

Stem cells provide revolutionary advancements in the treatment of severe conditions such as blood disorders, autoimmune diseases and certain cancers. Haematopoietic stem cell treatments have been proven to benefit patients living with these conditions. However, a previous lack of regulation has meant some private clinics throughout Australia have been offering what they claim to be stem cell treatments for a wide range of conditions without evidence that their approach works or is safe.

New regulations from the TGA that came into effect on 1 July 2019 are attempting to protect consumers from the potential harms of unproven treatments, while maintaining access to well-established therapies.

Stem cell treatments involve the use of unspecialised cells—stem cells—to repair or replace other cells in the body that are lost through disease or injury.

While stem cells hold great promise for the treatment of many currently incurable and debilitating conditions, development of novel stem cell therapies is still in its infancy for most diseases. More research is needed to establish the risks and benefits before such new therapies should become widely available and patients are charged for the treatment.

Under the new regulations, which expand the TGA’s oversight of therapeutic products derived from stem cells, all non-hospital-based providers must now comply with the TGA’s safety, quality and effectiveness requirements, even if the cells are obtained from a patient for their own use. This means that providers who continue to provide unproven stem cell treatments may face criminal penalties.

Welcoming stricter regulations

Associate Professor Megan Munsie, of the Department of Anatomy and Neuroscience, welcomes the new regulations. In her roles as deputy director of the Centre for Stem Cell Systems and head of engagement, ethics and policy at Stem Cells Australia, Associate Professor Munsie has campaigned for stricter regulations for the past eight years.

“It’s very necessary for the TGA and others to take a strong stance here, so the demarcation of what’s acceptable and what’s not is not just left to the patient to try to navigate,” she tells Medical Republic.

Associate Professor Munsie not only has extensive technical expertise in stem cell science, but also specialises in the complex ethical, social and regulatory issues associated with their use. Her laboratory at the School of Biomedical Sciences aims to improve understanding and awareness of these issues using empirical evidence, and to inform national and international policy around responsible innovation in this emerging technology.

Stem cell cultures under the microscope.

Loophole concern

The decision in the new regulatory framework to broadly exclude products made from the patient’s own cells and tissues from TGA oversight, if this occurs in a hospital by a doctor or a dentist, concerns Associate Professor Munsie.

“I can imagine that some hospitals may not be well equipped to explore the claims made by a company or practitioner who’s saying: ‘I’ve been doing this for years, look at my portfolio of patients and fabulous results’.

“I think the TGA are trying to address this problem as best they can, but we’ll need to keep monitoring to make sure it has the effect of protecting patients from possibly dangerous and exploitative practices.”

There have been several reports of adverse outcomes from stem cell interventions from around the world, including cancers, infections, strokes, cardiac arrest, blindness and even death. A women died in Sydney following complications of a stem cell procedure to treat her dementia. The risks of stem cell interventions depend on the type of cells used, their intended use, and how they are manufactured and administrated. Just because the patient’s own cells are used is no guarantee that the treatment is without risk.

While the new regulations are unable to protect patients seeking access to unproven stem cell treatments overseas, it is hoped that the they will help prevent such devastating outcomes for Australian patients in the future.

“The staff I’ve worked with at the TGA are trying,” says Associate Professor Munsie. “After eight years of asking for change I’m thrilled that someone is listening. It’s progress.”

Read more about the new regulations and Associate Professor Munsie’s response here.