Unproven cell therapies: perspectives on regulation and education
At the 2023 Annual Meeting of The International Society for Cell and Gene Therapy (ISCT; 31 May-3 June; Paris, France) we spoke with Laertis Ikonomou about the issues surrounding unproven cell therapies. Laertis gives us his perspective on the evolving field of cell therapy regulation and explains how we can better equip patients with knowledge of the risks associated with unproven cell therapies.
Please introduce yourself
My name is Laertis Ikonomou. I’m an Associate Professor of Oral Biology and a member of the Center for Cell and Tissue Engineering at the University at Buffalo (NY, USA). I’m also the current chair of the ISCT Committee on the Ethics of Cell and Gene Therapy until the end of this meeting when I’ll become the immediate past chair.
What are the main issues around unproven stem cell therapies?
I think the main issues are the same issues we had 15 years ago. Although the landscape is changing in terms of the interventions offered, I think the core issues lie in people using ill-characterized preparations and administering them to patients without any evidence of safety or efficacy. This lack of established safety and efficacy profiles are a significant concern.
Many businesses in this industry also have direct-to-consumer advertising of their offerings. It’s a major characteristic of this industry and that’s why unproven stem cell therapies are so able to reach large numbers of patients. Additionally, many of these businesses are willing to fight back when regulators try to contain the industry. We’ve seen that happen in the United States with legal battles between the FDA and certain businesses.
How much of an impact does regulation play?
Regulation alone is not enough. This is a very complex problem and these symptoms didn’t arise out of nowhere. Rather, it was kind of an organic phenomenon that took place over several years, aided by several factors.
The first problem is that there is a demand for cell therapies and when we cannot offer approved therapies businesses will step in and offer unapproved therapies.
I think the other problem is there is a very low barrier to entry in many regions for those businesses offering unapproved therapies. Even in regions with strict regulations in place, such as the United States, enforcement remains a challenge. Businesses can easily switch from one unproven product to another to evade regulatory scrutiny. With a vast marketplace and numerous players, enforcement becomes increasingly difficult. It’s not merely about having regulations; it’s about effectively enforcing them.
If regulation alone is not enough, how do we tackle these issues?
Well, we need several different approaches and strategies. First of all, we need to engage a lot of professional associations, medical societies, scientific associations and most importantly, patient foundations and groups. Because these are the people who can influence their peers. People will go and seek out these unproven cell or cell-based interventions. But if they a network of peers that will inform them and try to discourage them from seeking out unproven treatments, I think that will be very important.
On one front, regulatory agencies like the FDA, the EMA and TGA, can work to regulate and contain the marketplace. At the same time, we need to inform and educate patients of the risks and the harms associated with unproven cell therapies. These risks also go beyond physical harm. They include psychological and financial repercussions. it’s essential to raise awareness within the community about the lack of revolutionary benefits and the significant risks involved in these treatments.
How do we ensure patients are adequately informed?
I think we should develop more condition-specific resources. For example, when a patient with a certain condition is seeking out therapeutic options, they should readily find information explaining that experimental drugs do not have proven safety and efficacy. I acknowledge that this may not always be straightforward because some people will seek out these treatments no matter what. But I think this should be a concrete step. We have targeted campaigns to develop educational materials where people can get up-to-date, validated information about what is available as a therapeutic option.
Secondly, we need to be constantly bridging the gap between scholarly literature and public knowledge. There are people in the field doing amazing amounts of work and producing a wealth of quantitative data about the effects of unproven therapies, but that doesn’t reach the public. So, I would say part of this awareness campaign should involve reaching out to the lay press who cater to a broad audience and can help convey these messages.
From my experience in the United States, we are witnessing an increase in investigative journalism shedding light on these businesses and their practices. These journalists often consult with academics and researchers, and I think the message is coming out little by little.
What has been your favorite part of ISCT?
My favorite part, because I’ve been highly involved in them, is the roundtables. I have moderated two of them and then I was a panelist in two others. I think it’s a novel format. It was introduced at the last conference, which unfortunately I missed. But this year I’ve been in four of them and I find it to be a great format.
Disclaimer
The opinions expressed in this interview are those of the interviewees and do not necessarily reflect the views of RegMedNet or Future Science Group.