Regenerative Medicine 3.0 : Is it real this time?

November 4, 2015 ctheodoropulos

Cell therapy is back and bigger than ever. In the mid-1990s, companies raised hundreds of millions of dollars to fund the promise of cell therapy, noted Mike Ward, Global Director of Content for Informa Pharma Insights. “And it went nowhere. Literally billions of dollars were spent back then, and just as quickly it all disappeared.”

Today we are again seeing some very big deals with large price tags backing the renewed promise for cell therapy, he said. “All this is clearly exciting, but we have been here before.

“Why should we believe it is different this time?” he asked, welcoming a panel of executives invited to address the practical issues behind the hype in cell-based therapies at BIO-Europe® 2015 in Munich.

Michael Leek, left and Philippe Duchateau.

Michael Leek, left and Philippe Duchateau.

The CEO of TC BioPharm, Michael Leek was equal to the provocation, acknowledging the weaknesses in early cell therapy programs, and promoting the strengths in this new wave.

“It was a brave new world in the mid-1990s, one where we didn’t really understand the mechanisms of action, the effects of the therapy, nor whether those effects would be sustained,” he said. “If you look at products being developed now, we understand very well those mechanisms for very focused, very directed products. Clinical trials have been designed with a rigor. Cell Therapy 3.0 has a much better chance of working because we are thinking about it more intelligently than we did back then.”

The Chief Scientific Officer at Medigene, Dolores Schendel added, “We have also gained two decades of knowledge about the mechanisms of the immune system. We have additional tools that help our cellular therapies be more potent. We have clinical success, and we are building on that.”

Philippe Duchateau, the Chief Scientific Officer at Cellectis responded in one worddata. “It all went down before because the data was not there. It is coming back because the data is there,” he said.

It was then the turn of the panelists to frustrate the moderator.

Leek said that moving forward, cell therapy will depend critically on how the business is “pharmaceuticalized. Having a product with a six-hour shelf life is commercially difficult, so that how we get a product from a clean room into the patient is where academia, pharma and biotech need to work to create a new set of logistics to make these products more pharmaceutical.”

Everything depends on the nature of the cell therapy, he explained. Where it is autologous cell therapy, the patient is the batch and the product is a service. But to scale out production to serve the entire target population, a pharmaceutical company would need a clean room the size of Glasgow.

Where it is an allogeneic from a universal donor, then it becomes a product that can be stored in any hospital pharmacy anywhere in the world.

“Is it a product or a service, then?” Ward asked, setting off a roundtable debate about the virtues and challenges for both approaches, and the potential for a service model that could be offered by non-pharma players, such as medical device companies.

A question from the audience proved to be even more provocative than the questions from session moderator Ward.

With more than USD 10 billion invested to date in CAR-T (chimeric antigen receptor) programs, a day of reckoning is coming sooner than companies may be prepared to face, according to the executive posing the question. If companies do not deliver, he said, if production and cost issues are not solved, if no one is able to turn this into a pharma business, the air is going to go out of the balloon.

Duchateau responded that as long as clinical data matches people’s expectations, the sector should be fine.

Leek agreed that there is “an amazing CAR-T bubble and some would not ask if it will burst but when it will burst. If and when it does it would take the breath away, yet not all cell therapies are CAR-T. It will make it harder for people to invest in cell therapy generally. But I do not think we will be dead in the water.”

Which allowed Ward to conclude with a statement he clearly had waited an hour to deliver, and said he could not longer resist saying, that “Cell therapy isn’t going to died in a CAR crash.”

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