10-12 years ago everyone was talking about cell therapy and regenerative medicine being the next big thing, and the hype was through the roof. Yet, somehow it didn’t explode, and what we’ve seen is a steady, but slow, advance.

Now, the hype is back. So, is it justified?

I was fortunate enough to be invited to attend the Alliance for Regenerative Medicine’s EU Advanced Therapies Investor Day, and spoke to several of the CEO’s to get the skinny.

I asked Eduardo Bravo, CEO of TiGenix about whether the current hype was justified. If anyone would know, he would. TiGenix was the first company that got a cell therapy approved under the new legislation in Europe way back when - and now, more importantly, they just announced in August the first Phase III data with stem cell treatment.

Here’s what he had to say:

Eduardo: I think when the market started a little over 10 years ago people were excited, and there was a lot of hype that these stem cells would be the cure for everything. The problem is, of course, that it takes 10 years to develop a compound! It has taken those 10 years to secure the necessary investments, enhance our learning about the cells, and do the clinical trials.

We’re now coming at the time when those well controlled, large well run clinical trials are starting to be presented. And 601 is a great example - that’s the first one with positive data. I’m pretty sure cell therapy will be a revolution. We’re at the very beginning, but the data is coming, and people will start understanding how we can use it better and better.

It’s all about the data…

“The data will define the hype”
Zami Aberman, CEO, Pluristem

Zami Aberman, CEO of Pluristem, an Israeli biotech that produces placenta-based cell therapies and its indication for critical limb ischemia, also highlights data as the real driver:

Zami: I don’t know when the revolution will really take off: one year, maybe two. The only real driver will be good clinical data. TiGenix is the first to start. Let’s hope others present good data. Then the pharma companies and investors will realise it’s real.

Eduardo Bravo was also very keen to point out that data is the fundamental driver and catalyst to the success of cell therapies:

Eduardo: I think it is all about the data. If we can provide exactly as we’ve done, with real, meaningful data on large controlled clinical trials, then people will be convinced.

The good news is there are a number of trials that will be coming out soon, with very high hopes of success.

Pharma acceptance

Critical to the success of cell therapy and regenerative medicine will be pharma adoption, interest and investment.

Zami mused that if one of the pharma companies adopts cell therapy as the next generation biological product then everyone will follow. 20 years ago, he pointed out, all the biological antibodies were questionable. Today each pharma company is running after that.

There is a real sense that it will be the same with cell therapy.

Zami believes that one of the reasons why we are now at an inflection point, is precisely because pharma is beginning to take notice. As Zami says:

“What is driving excitement now in cell therapy? Two factors: Firstly, we have now had significant clinical outcome. And secondly, we have seen the acceptance of the large pharma companies”

Zami Aberman, CEO, Pluristem

Data is clearly a driver of pharma acceptance, but so is one other factor: the success of immunotherapies.

Immunotherapy: Game Changer and Precursor

Immunotherapies have rocked the life science industry in the past few years, The technologies have fundamentally redefined the sector, generated significant investor enthusiasm and shifted the focus of pharma’s strategy.

But one of the most significant things it has done is establish a precedent for an impending cell therapy revolution. The success of CAR-T and immunotherapies is acting as a driver of investor and big pharma interest in cell therapies and regenerative medicine technologies, and is setting the stage for the take off of cell therapies.

For example, here’s what Kieran Murphy, President and CEO, GE Healthcare Lifesciences had to say when asked what was most exciting him about cell therapy at the moment:

Kieran: Progress with immunotherapy in cancer clinical trials. The field is reaching a level of maturity at the moment that indicates it is clearly at an inflection point, and what’s most exciting is that, while we are seeing real curative successes, big pharma has really engaged solidly with the field.

Zami of Pluristem pointed out the link between immunotherapy success and cell therapy success:

Zami: Immune therapy or gene therapy was dead for about 15 years. Yet now Immunotherapy in cancer treatment has made huge progress, Pharma companies began to realise the potential and now everyone is entering into immune therapy.

The investment community is also realising that the immune therapy revolution is proof of the principle that cell therapy is the next generation. Now they just have to decide on which company they are betting.

Regulatory authorities are keen

“Regulators have really understood the requirement of this industry and taken action to make the development smoother.”

Stephane Boissel, CEO, TxCell

Another reason for optimism is that the regulators on both sides of the Atlantic appear to be very keen, and the CEO’s I spoke with were singing their praises.

Stephane Boissel, CEO, TxCell, a cellular immunotherapy company that is the only platform company in the world using exclusive regulatory T-cells said:

Stephane: Now when we talk to the regulatory authorities you have people who truly understand our challenges, and we are not treated as a biologic developer or developer of a new clinical entity. It’s a totally different approach, and we are very glad that the regulatory bodies on both sides of the Atlantic, (as well as Japan by the way, where they changed regulation not that long ago) are keen to really accelerate development in this space.

We are very grateful that regulators have really understood the requirement of this industry and taken action to make the development smoother in that respect.

BUT, key challenges remain:

While the cell therapy revolution appears to be on the cusp, it is important to stress that the sector still has significant challenges:

More good data needed

While we have seen some good data already come out, clearly the jury is still out. In the next year, we should see the emergence of the data that will, as Zami says, ‘define the hype.’

Reimbursement and Pricing remains most significant challenge

‘The biggest challenge will be reimbursement’
Stephane Boissel, CEO, TxCell

No surprise here. In order for a therapeutic product to be successful, it must be profitable! So, Stephane – like most at the ARM event – is concerned about reimbursement and pricing:

Stephane: Probably the biggest challenge will be reimbursement. We’re going to be treating mainly one of those diseases that are today treated chronically. So, how do we ensure that we are properly rewarded? And how do ensure that the authorities maintain a long-term mentality of the savings that these therapies provide? This remains to be seen.

Commercial successes still needed

Success breeds success, and nowhere is this truer than in pharma. As Eduardo put it:

Eduardo: Because we’re still in infancy we really need a couple of commercial successes - so people realise that not only can the cells do what they need to do but companies can make money!

Confidence and collaboration

So, what are the ingredients needed to cross the line?

Confidence – of patients, investors, regulators and pharma companies – and collaboration is the biggest catalyst for success

Kieran Murphy, President and CEO, GE Healthcare and Lifesciences

Kieran Murphy of GE believes he knows:

Kieran Murphy: Confidence – of patients, investors, regulators and pharma companies – and collaboration: no one can do this alone – it is going to take big pharma, niche biotech, government, regulators, researchers, clinicians, healthcare systems and tools and technology providers to work together to address the challenges and effect the systemic changes needed to bring the promise of these therapies into widespread use. Fortunately, that appears to be widely understood and acted upon.

I’m compelled to agree.
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For cell therapy the next milestone that the industry is expecting is the first registration or licencing of a product. It could happen in 2017. And that should again really mark the start of a new era for the sector.

We are only at the beginning of something that could really become a therapeutic field in itself, but it’s clear that cell therapies are entering a new era – one where the hype might well be justified.

The investment community is beginning to realise this, and as Zami said - now you just have to decide on which company you are betting.