Manufacturing It’s not all CAR-T cells
Although they tend to dominate the conversation due to the leaps in research and positive clinical applications they’ve had, CAR-T cells are one of many cell and gene therapies. “CAR-Ts have the limelight right now,” says Kusena. “Other therapies aren’t getting as much because there isn’t as much evidence to support them.” Kusena’s own work during his PhD was centred on dopaminergic neuron progenitors – a stem-cell derived therapy with the potential to treat Parkinson’s disease. “That’s basically taking embryonic stem cells differentiated into neurons and then implanting them into patients,” he says. Bluerock Therapeutics, a subsidiary of Bayer, is also conducting research into the viability of these cells as a treatment for Parkinson’s, and it had an Investigational New Drug (IND) application cleared by the FDA in January. Although most of them are CAR-T cell therapies, there are a few FDA-cleared products that target other maladies. One of these has been cleared for clinical use in the US since December 2016. Maci, an autologous chondrocyte implantation therapy, is used to replace cartilage cells in the knee to repair damage. Another osteo- related therapy approved by the regulator is Zolgensma, a single-dose treatment for spinal muscular atrophy, which kills 90% of untreated infants before age two. The $2.47m price tag on Zolgensma drives home the challenge inherent within the field of cell and gene therapy. Although the full list of approved FDA products runs to 22, with many more expected to be cleared in the near future, unless manufacturers find a way to scale up production, the cost will remain prohibitive.
manufacturing side of the industry. But in the US, most companies still require applicants to have a four-year college degree in chemistry or biology, so that they come to the manufacturing facility with a basic understanding of laboratory science. “If somebody comes into a cell-processing lab and they don’t know how to use a microscope, they’re going to have a hard time finding a cell,” says Janssen. But despite this, he believes a potential solution to the skill shortage is casting the net for applicants wider and being more willing to provide training to those who haven’t stepped foot in a lab. “It’s conceivable that somebody with the critical thinking skills required for getting an undergraduate degree in philosophy, with a little extra education, could be trained to do cell and gene therapy manufacturing,” he says. One avenue Janssen believes could be used to provide this additional training is community colleges, and he says there’s already ambitions within the industry to form partnerships with them. “Desperation breeds enthusiasm,” he chuckles. “The companies
are certainly realising they’re going to have to invest in this area.”
A long shot
Despite the undeniable benefits of automation, the move from manual to machine-based processing might not be as smooth as some anticipate, and Janssen says that’s due to the need for another form of expertise that’s in short supply – process engineers. “There’s going to be a need for process engineers who are versed in how cells grow and how gene vectors enter cells, as well as the things that inhibit the efficiency of both,” he says. “I already know that there’s a shortage of such folks.” On the manufacturing side, there’s still hope that the skills gap will be closed, but not an awful lot of belief that it will. “It’s going to be at a steady state in the same position we’re in now for a while,” says Stoll. Kusena is more optimistic about the UK’s cell and gene therapy sector, having seen an uptick in the number of people coming into the industry without a PhD, but instead with training provided through programs like CGT Catapult’s apprenticeships. While it used to be the case that PhD educated staff were necessary to understand the process and make changes to it, he says “now, the more it’s being standardised, the easier it is to write a standard operating procedure that somebody can just follow, with a senior supervisor there to respond to more technical issues.”
Janssen, who describes the subject as being “near and dear to my heart”, hopes to see the industry embrace students from outside of science in the US too. For the moment, he concludes his thoughts on a sombre note: “The number of people who could benefit from CAR-T therapy is probably somewhere between ten and 50-fold greater than the number who currently have access to it,” he says. “If we cannot disseminate these kinds of therapies to a larger population, we’ve really not accomplished all that much.” ●
The demand to produce vaccines at scale has created more competition for skills needed in other areas of cell and gene therapy manufacturing.
38 World Pharmaceutical Frontiers /
www.worldpharmaceuticals.net
solarseven/
www.shutterstock.com
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