The Building Blocks of a Better World: Cell Therapy and The Changing Landscape of Regenerative Medicine

The Building Blocks of a Better World: Cell Therapy and The Changing Landscape of Regenerative Medicine


Alain VertesA conversation with Alain Vertès, Director of the EU Section of the Alliance for Regenerative Medicine and Managing Director of NxR Biotechnologies.

When asked for a story that illustrates the power of cell therapy, Alain Vertès looks to the story of his cousin who recently died of triple negative breast cancer, despite an early diagnosis. “I believe that if there had been [cell and gene] therapeutic products that enhanced the properties of her immune system, that was more personalized, it might have changed things.” The potential of cell therapy, a potential that includes innovative therapies for patients with today’s incurable diseases, is what drives his passion.

Vertès is Director of the EU Section of the Alliance for Regenerative Medicine and Managing Director of NxR Biotechnologies where he consults in business development focused on biotechnology deployment and innovation funding and commercialization. He has worked as a researcher and has had hands-on involvement in the advancementof radical innovation, including leading global therapeutic stem cell initiatives. This week, Vertès will take part in the Alliance for Regenerative Medicine Investor Day conference in London, England organized to provide institutional, corporate and private stakeholder insight into the investment hypothesis for regenerative medicine. In anticipation of the conference, the following is a conversation with Vertès from his home in Basel, Switzerland.

The Building Blocks of a Better World: Cell Therapy Is Changing the Landscape of Regenerative Medicine.

What does the average consumer need to know about cell therapy?

The first point is that cell therapies are rapidly emerging as potentially curative, individualized treatments for many of the world’s most challenging diseases, especially inflammatory diseases and cancer. In oncology, these therapies are programmed in a way that a patient’s own cells attack cancer cells, but it is critical to bear in mind that there are various versions of cell therapy to fight various diseases. These cell types range from mesenchymal stem cell, engineered hematopoietic stem cells, pluripotent stem cells derived cells and tissues, and engineered cells of the immune system, for example chimeric antigen receptor T cells and NK cells.

To make a long story short, we have different products with different therapeutic properties and different challenges; but all of these products leverage the disease-fighting capacity of the human body. It is also remarkable that by combining these cells with other types of medicine, we are able to enhance this inherent disease-fighting capacity. The combinations of CAR-T cells with small molecule checkpoint inhibitors, for example, might be the key to unlock the path to treating treatment resistant solid tumors.

What led to your involvement with the Alliance for Regenerative Medicine (ARM)?

First and foremost, my passion for this new field. I would say, first, I am a researcher—microbiology and molecular biology—and then of course a business career: I am a Sloan Fellow from London Business School and have worked in business development for most of my career.

The Building Blocks of a Better World: Cell Therapy Is Changing the Landscape of Regenerative Medicine.

If you think of the impact that the technology of monoclonal disease therapy has had in current health care—that was twenty-five, thirty years ago, right? We can sense a similar impact of cell and gene-based therapies emerging.

It’s hard to realize the potential of the new therapeutics. Likewise it was very difficult to understand the impact of monoclonal therapeutics in its time. Let’s not forget that the first fifteen clinical trials of monoclonal therapies were a total failure; and the only trial that went through after the first batch of fifteen was a commercial failure. What I’m trying to say is it’s hard to understand today, the positive impact new technology can have. That’s why I’m fascinated. We are in the position to accelerate this field. We are in a position to write pharma’s history—again.

What is needed to push radical innovation is to create a forum for networking, the sharing of ideas; more education and communication; better assessment of technology and commercial risk to trigger more investments; engaging people and policy makers, and pulling people in the same direction to continue to build this new field; and of course using the collective wisdom to draw up the best strategy for this field—for health care in general, really, and for the ultimate benefits of patients worldwide.

ARM provides this linking between different actors, different contributors, in cell and gene and immune-based therapies. It’s a good place to work to try to impact and accelerate the process.

It’s a way to contribute to making a better world.

The Building Blocks of a Better World: Cell Therapy Is Changing the Landscape of Regenerative Medicine.

What can the cell therapy industry do to achieve the goal of making cell therapies available at a faster rate to healthcare?

Communication and education—that’s really important. It’s very important to reach community acceptance of new products and to make sure ethics are not forgotten along the way. What’s more, the industry should make sure that it is adopting and understanding the promise of the new technologies, as well as its limitations, as well as communicating those dimensions to the general public. And we need to have more engagement of pharmaceutical companies, because these companies can help accelerate the field with their exquisite capabilities in incremental development and commercialization.

So, more communication and education, the adoption of the technologies by more of the large players, more financing, which is important, and also to emphasize the issues relative to commercialization as soon as possible. We need to ensure that a virtuous circle of investment is created: building shareholder value is great for patients because that means more unmet medical needs will be met in the nearer future. That includes manufacturing, that includes logistics, and that includes pricing and reimbursement, which is a topic that nowadays comes up time and time again at conferences given that we already have curative medicines being launched.

The Building Blocks of a Better World: Cell Therapy Is Changing the Landscape of Regenerative Medicine.

We still have to identify what will be the best price and reimbursement model for curative therapies and nobody really has a satisfactory answer so far. That needs to be looked at in collaboration with all of the stakeholders: patients and physicians, hospital administrators, pharmaceutical companies, biotech developers, payers, governments, regulators. We have to make sure that we have a dialogue relatively quickly—to avoid developing [cell and] gene therapies that are life-saving but launched at such a high price that you end up with a market of just a handful of patients. Essentially, I believe in developing these products for the greatest numbers, and they will impact healthcare if they are affordable…while preserving profit margin incentives for developers.

So, keeping an eye on affordability while at the same time having enough money to be innovative?

It’s an important balance. We want companies to be encouraged in developing, we want investors to be encouraged in investing, and we have to consider that investment makes sense if money put on cell-based or gene-based therapies allows the company to make as much money on their investment as if they were putting it into something totally different, including, for example, a commodity product in a totally different industry: an important economic mechanism at play here is opportunity cost: we need to ensure that money invested in regenerative medicine will provide a yield that is no lower than if it were invested in a conventional therapeutic and even in a totally different industry altogether..or simply in bonds.

At the ARM conference next week, which session are you most looking forward to?

All of the sessions we have are about critical issues. I’m chairing one panel on cell therapy manufacturing. Manufacturing is obviously very important to bringing the product to the marketplace. If we can decrease the cost of the therapeutics and ensure highly efficient logistics, and if it’s possible to offer less expensive therapeutics to patients—that would be a tremendous achievement because one could decrease the overall burden on the healthcare system and payers.

Then again, it’s very important to find that fine balance. Manufacturing logistics are critically important; that being said, the world conference (ARM) is about investors, which are providing critical fuel for the regenerative medicine industry. We need to have more investment, and investment that is more sustainable, in order to get more options, more products—and thus more patient benefits—into this field.


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