How cell and gene therapies are infusing blood centres with new purpose

As the cell and gene therapy industry grapples with infrastructure challenges, help is emerging from an unlikely source – blood donation centres. Expanding beyond their traditional roles of collecting blood and plasma for medical treatments, these centres are starting to leverage their technology and trained staff, as well as their ties to local communities, to support the next frontier in chronic disease treatment.

New advances are being made every day in cell and gene therapy, and regulators expect to clear as many as 17 new cell and gene therapies this year, with more coming each year after that. However, developing these therapies and getting them through the FDA is only the beginning. Cell therapies face myriad challenges when it comes to actually getting them to the patients that need them most – not only on the reimbursement side, but also logistical challenges.

“A lot of these patients in the autologous realm have to travel very far to get to a collection site or centre, to even be apheresed, and to then travel back weeks later to have that material reinfused into them once the therapy is manufactured,” Priya Baraniak, chief business officer at OrganaBio, told Deep Dive. “And if you think about these patients, this is their usually third or fourth line of therapy. They’ve already gone through chemo, radiation, maybe a monoclonal antibody, you name it, and they’re highly, highly compromised.”

And right now, the number of sites qualified and accredited to administer cell therapies is shockingly small.

“There’s a limit on the number of what are called immune effector cell accredited hospitals in the country. There are like 130 of them out of 5,000,” Becky Cap, who at the time of this interview was SVP Business Development at BioBridge Global and now works at Vitalant, told Deep Dive. “And if you then look at the number that are NCI-designated comprehensive cancer centres, that’s even fewer. So, we have to figure out ways for smaller hospitals – and I don’t mean the true community rural hospitals, but the hospitals in big cities that aren’t part of those designations – how do we either get them that designation or expand their ability to treat a broader number of patients?”

A lot of the problem-solving in the industry is about how to administer this therapy in more places – not just in few- and far-between academic medical centres, but in community hospitals and, maybe someday, even at the bedside.

A solution waiting in the wings

Meanwhile, there are blood centres. Non-profit organisations like the Red Cross, Blood Centers of America (BCA), BioBridge Global, and Vitalant showed up on the scene long ago to solve a similar problem – how to collect enough blood on a national level to support lifesaving transfusions, and then get that blood safely and promptly to where it’s needed the most.

“The use of blood by hospitals is the very earliest form of cell therapy,” said Lee Buckler, SVP of advanced therapies at BCA. “The bulk of the cell therapies, almost all the cell therapies that are commercially approved, are somehow derived from blood. It makes perfect sense that people who know how to harvest blood, process blood, store blood, keep blood safe in the medical supply system, are playing an active and important role in those cell therapies.”

It’s not directly comparable to cell therapy, but crucial components are there, including a localised, community-based infrastructure serving all hospitals, apheresis equipment and staff trained to use it – often designed to be taken on the road to blood drives in schools, churches, and libraries – and a safe, regulated supply chain to get blood from collection to delivery.

In the early days of cell therapy, Buckler explains, manufacturers weren’t thinking about blood centres because they lacked certain crucial facilities, such as clean rooms and cell engineering equipment. But as the field has industrialised, there’s been a shift in priorities.

“There’s this fascinating sort of trifecta, if you will, of things happening which have brought blood centres back into the equation,” he said. “First, it’s that automating and simplification of the machines used to manufacture these products, which look a lot like the machines used to process blood. Second of all, many blood centres have built clean rooms to put such machines in. And then thirdly, the therapeutic developers themselves and the clinicians recognise that the hospitals that they need to move into in the Mobile, Alabamas and Tulsa, Oklahomas of the world, don’t have the same kind of infrastructure that they do at City of Hope and Memorial Sloan Kettering.”

This is more than theoretical at this point, Cap says. While there are still kinks being worked out and a lack of awareness among therapeutic developers, there’s also already a lot of adoption.

“It’s really about making sure that the therapeutic developers know the capabilities of the blood centres, and the blood centres get comfortable with the specifics of what is being put out there,” she said. “But I can easily name half a dozen larger organisations that are really committed to this space, and they have a pretty significant footprint across the country. We still have a few things to work out for it to be smooth across the board, but some of the larger pharma companies are already working with the blood centres to make collection and delivery more efficient.”

Manufacturing and allogeneic therapies

While the equipment and training that blood centres have can help them potentially set themselves up as part of the infrastructure for autologous therapies, many blood centres are also looking at collecting biological material for allogeneic therapies – where patients would receive an infusion of modified donor blood rather than their own.

There are a few advantages here. Even though immediacy of location is less crucial than it is with autologous therapies, it’s still the case that transplanting blood and cell products long distances is complicated and expensive, so shortening those distances is a positive. Additionally, selling biologic materials to pharma companies is a potential revenue stream for blood centres, which, even though they’re most often non-profits, still need to sustain themselves.

“To the extent that there is a revenue or operating surplus motivation, it goes to the sustainability of the organisation,” said Cap. “Because if you don’t know this, blood centre operations right now, typically a 3% margin is considered good performance.”

Chetan Makam, managing director at Terumo, which supplies much of the equipment blood centres and cell therapy developers use, agrees.

“If you look at how the whole blood industry works, our raw material is unreliable at best because we rely on the good-heartedness or the good nature of people to voluntarily donate blood,” he said. “I think no other industry or group of people would ever say this is a smart business, because you’re relying on the largesse of the human beings to donate. So, yes, people need to operate, and they need to make money to keep this whole system going. But I think they also see it as an additional way of helping.”

New challenges in soliciting

Speaking of good-heartedness, one difficulty of collecting biological material for allogeneic cell therapy is that it’s a different sell for donors. With cell therapies going for $2 to $3 million per treatment, people are bound to wonder why they’re giving away their cells for free.

“Part of where blood centres or not-for-profit organisations run into some barriers in this realm is that they do not compensate their donors,” Baraniak said. “When […] the Red Cross is calling on you to donate a pint of blood, you know that pint of blood is going to be transfused into an individual who has suffered some traumatic injury or has some sort of disease. There is, an altruistic piece of that. It’s much harder for people to grasp that in the realm of allogeneic therapy, where I’m donating something that might go to research, I’m donating something that is a piece of a therapeutic that might be developed, and maybe someday it’ll save thousands of lives, maybe it won’t. It’s a lot harder, I think, to recruit those individuals and keep them engaged for that sort of donation if you’re not compensating them for their time.”

On the other hand, Baraniak added, the skill of cultivating a donor pool is non-trivial and extremely important for the success of the industry – and it’s something at which blood centres are well-practised.

Finally, there are additional concerns when it comes to bone marrow donation and mobilised blood donation (where patients are induced to create extra white blood cells before being apheresed).

“In that case, you have two times in your life that you can be mobilised as a blood donor,” Baraniak said. “Shouldn’t those two times be used if you need it for yourself or a loved one needs your blood donation, rather than calling you in to get a leukopak for some researcher to put into culture in a lab and then dispose of that material? The ethics around it are quite fraught. I don’t know what the right answer is, but we do believe that on the adult side – if donors are coming in and making that donation – they do deserve something for that time.”

The future ecosystem of biologics

One thing Cap, Baraniak, Buckler, and Makam all agree on is that the cell therapy delivery groundwork that’s being built is not going look like one single entity solving all the problems.

“I think what it shapes up to be is an ecosystem, right?” Makam said. “The way I think of the ecosystem is, where do you start? How does this whole journey progress until you bring it back to the patient who’s at the centre of all this? Where is the delivery of the treatment being done? Is it at a hospital? Is it at a blood centre? Is it at the bedside of the patient at home? Don’t discount that – I mean, we’re doing dialysis at the bedside these days. How are we keeping track of this cell, so that you don’t make mistakes? Who’s carrying this? Cryogenics, logistics, all these other pieces that surround this ecosystem? So, I look at this evolving as an ecosystem with multiple partners that all work in conjunction.”

Baraniak agrees that the need is so great right now it will take many players working together to realise this ambitious goal.

“I think that it is going to be a concerted effort by everyone in this industry who has capacity and capability in any way, shape, or form to move this industry forward,” she said. “And we have to find ways of standardisation. We have to find ways of speaking the same language and working together collaboratively to really help patient outcomes. We’re going to have to find ways to leverage each other’s infrastructure and say, I’m on the East Coast, you’re on the West Coast. Here’s where the patients have the need. How do we come together quickly and make this happen?”

Luckily, that’s something else that blood centres have been doing for a long time.

“While each group has a desire to be a leader, a winner in its own right, our goal is to make sure that the healthcare needs of the nation are met,” Cap said. “So, if you think about the blood model, we actually have networks of emergency call chains. If there’s a disaster in Florida and the blood bank can’t collect or it’s without power, there is an automatic call that goes out to the other blood centres around the country, and there’s a commitment of number of units of blood and blood products that each one will commit to send to that disaster area. So yeah, we’re competing until there’s a crisis, and then we call it co-opetition.”

Cell and gene therapies are poised to transform medicine in dramatic ways. But even if cell therapy is a brave new world, it’s going to take the old players, acting – and interacting – in new ways, to make it a reality.

Jonah Comstock is a veteran health tech and digital health reporter. In addition to covering the industry for nearly a decade through articles and podcasts, he is also an oft-seen face at digital health events and on digital health Twitter.

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