By Erin Harris, editor-in-chief
In part one of my discussion with Miguel Forte, CEO at Zelluna Immunotherapy and Chair of Commercialization Committee at ISCT, he answers my questions about academic-to-industry translation, the root cause of unproven therapies, how the industry is combatting them, and more.
Harris: At Penn Medicine’s Business of Regenerative Medicine: Innovation, Clinical Translation, and Entrepreneurship event this summer, you gave a thoughtful presentation on academic-to-industry translation. You explained that the cell and gene therapy industry is in its “teenage phase.” Explain what you mean by this.
Forte: ; Getting to the adolescent years, an adolescent has already done things, has already established its presence, but is still going through the growing pains of the challenges establishing itself and going beyond, and really defining the personality and the space it’s going to occupy.
We’re seeing that with cell and gene therapy. A few years ago, we were considered as cute. Like, “Oh yeah, there’s probably some potential.” Today, there are clearly already some successes established. There are several products on the regenerative medicine, on cell and gene therapy that have reached the market and with documented value being handed to patients.
But it’s only the beginning, and there are some challenges in establishing that — challenges in manufacturing, challenges in reaching the patients in adequate numbers, quantity, in being paid and reimbursed.
We have not really found the entire personality — a mature personality — a balance between the different players. That’s what we see. I’ll give you some specific examples. Take the more recent CAR-T products that have entered development from academia into industry, just over five years ago.
They showed fantastic value, fantastic promise, and eventually got to the market. But they’re still finding challenges in getting the right number of products produced and having some challenges in manufacturing, as well as being paid and reimbursed, as I mentioned, in the UK.
The indications for young patients have been supported by the indication for older patients, and a larger number of patients have been challenged in terms of documenting the competitive value to other existing alternatives. It’s exactly that.
The products are trying to find their place amongst the other opportunities in saying, “Look, we are now a player.” Cell and gene therapy is emerging; it is becoming an established field of new therapeutic opportunities, a new field in the medicine that people must now recognize and accept. It wasn’t before, but it is now, and it’s still growing and finding its own place.
Harris: Also during your presentation, you mentioned mentoring programs as an area of interest for cell and gene. Please explain.
Forte: I have two perspectives on this. First, my own personal interests, and my own personal experience in growing talent and developing talent across my career, particularly now in cell and gene therapy. But also, as an ISCT representative, because ISCT has a focus on developing talent and mentoring younger professionals.
If I take a step back, this is a new field requiring new expertise — expertise that needs to be anchored on what was there previously in the development of medicinal products. There are not a lot of people with experience in cell and gene therapy, in manufacturing, in developing this product in the clinic, and in bringing them to the market.
The lack of existing talent, the interest of new professionals has raised the need to how do we train them? Do they get trained only on the job, or are there additional opportunities to train them? Train them not only on a technical perspective, but also on a personal and behavioral and organizational perspective.
A lot of us will do that, when we hire younger talent into our organizations and grow them, and I have enormous pleasure and have successes on having done that. But ISCT as a program, particularly for young members that want to get attached to senior members in areas of their interest, either because they want to expand their knowledge and experience in that area or because they are working in that specific field and want to have a sounding board or someone that helps them.
At ISCT, we’ve established this program. There is a group of senior members that got together to find a program, and a group of young members that wanted to participate who then joined.
But there is an initial discussion with everybody together, and then there is a relationship mentor and an individual being mentored that goes on with opportunity for the young person or the person in training or the person in the growth opportunity to reach out, to have a discussion, to have a sounding board.
The areas where most of this is happening relates to areas of manufacturing, but also some areas of research and some areas of pre-clinical development more than necessarily on the clinical aspect, where I would say that all practices are more and more established.
It is a combination of recognizing the needs, recognizing the opportunity, and being very much up the street of the mission of ISCT, which is develop the opportunities to bring products, and the cell and gene therapy products, to patients. We see this to not only fulfill the expectations of young professionals but increase the impact of the field and, consequently, eventually deliver more value for patients.
Harris: Address and explain to us the root cause of unproven therapies and how the industry is trying to combat them.
Forte: I think the basis of the problem is individuals taking advantage of patients in need. Put yourself in the position of a patient that has a death sentence, because it got to the end of the opportunities of treatment, and they found there’s nothing else. They will be willing to find any opportunity, and some people take advantage of that eagerness to look for opportunities to solve an unsolvable problem and deliver an option that has not been proven, taking advantage of that weaker situation.
That’s why I feel very strongly, because it is taking patients in a weaker position in need, to find a solution and taking advantage of that. Now, there are multiple degrees of unproven. This is the medical tourism — the extreme of unproven cell therapy taking advantage of patients. There are also other elements.
There will be situations where unproven cell therapies are used for the first time, but if they’re used in the context of an assessment, a clinical trial, an expanded access or a single patient use, it’s yet unproven but nevertheless being tested. It’s the adequate context.
The first thing the industry needs to do is to assess unproven approaches in the context of the proper assessment and the proper clinical trial — not to be used in the context of taking advantage of patients in need.
There is a specific group within ISCT led by a past president who is producing a guide document that has been translated now into multiple languages, trying to educate not only the public at large but patients in need, saying, “Look, this is what you should consider when you’re assessing a new opportunity. This is what you should be prevented from doing. This is also what we should be doing as a society, and there are professional groups to avoid the incorrect use or the use of unproven cell therapies.”
As I said, this has been translated and published, and it can be on ISCT’s website.
I think it’s all our responsibility, of any of us that are involved in the field, to manage the expectations of patients by informing them of what are the opportunities in the clinical trials, conducting them to clinical trials, to give them the tools to assess and also give them that even some of the products that have been documented are in the market, and using cell and gene therapy, which is actually applicable to any medicinal product.
That can be used in a certain context, and it should not be used beyond that context. One thing that we’ve seen with some of these new CAR-Ts coming onto the market is patients believing that a panacea for a lot of conditions and approaching physicians on, “Can I have access?”
It’s important to educate the patient, educate the public at large, that these products provide enormous value when used in the right context for the right patient, and that’s what has been documented in clinical trials, which led to the regulatory approval. It’s important to understand and manage the patient’s expectations along the different portfolio of products that are now available.