Manufacturing
patient won’t react badly,” says Rafiq. “But if we use cells from a healthy donor, especially with T cells, there may be some reactions – so how do we edit them so patients do not have any adverse reactions? Once we can do this, we can start thinking about large-scale technology and using cells from one donor to treat lots of patients.” According to Rafiq, There are already clinical trials underway for the universal donor therapies, but we are at least five years away from them being a viable treatment. “The technology is already there and will mature over the next two years, especially as we start to see the outcomes from pivotal clinical trials,” he adds.
CAR-T cells have been a breakthrough in the world of CGT and have completely eradicated certain forms of blood cancer
learning to help us create a more consistent product.” It is not just the tools that could do with a helping hand though. “We need innovation around the automation of data handling, such as batch records,” says Rafiq. “If you are manufacturing at a clinical site then they are done manually, but this can take a long time and is open to human error, so it could be improved with data systems.”
The race to drive down costs Over the next few years, Rafiq believes some of these innovations in automation will open up the potential world of universal donor CGT therapies, which use T cells and natural killer cells from a single donor to treat many patients. “The platforms are designed for one patient, but the field is starting to move towards providing treatment for more patients, which will bring the cost down,” he says. “The more batches you can make from a single source, the more you can reduce the cost. It’s all about creating economies of scale so that we are able to move away from the patient-specific approach.” There is, however, quite a significant barrier of risk to overcome before the universal donor approach can bear fruit for patients.
“We are seeing different expertise coming into the field and we’re now starting to see the impact of overall deliverables. It’s a team endeavour rather than a solo pursuit. It’s very exciting.”
“With personalised therapies, we can be confident that when the cells are given back, the
42
One critical component to achieving all of the great ambitions laid out by Rafiq, including the automation of CGT manufacturing, is addressing the shortage of skilled workers in the sector. “There’s a shortage of highly-skilled individuals, which is becoming apparent due to the meteoric rise in the number of companies,” he says. “Ten years ago, there were just a handful of CGT companies. Now, there is in excess of 1,000. “It’s now a commercial endeavour, but it takes time to train people.” In response to the shortage, Dr Rafiq, along with his colleagues at UCL, recently created a master of sciences programme specifically geared towards this training – the MSc in Manufacture and Commercialisation of Stem Cell and Gene Therapies. He is also actively involved in international training programmes around the world looking to drive the industry forward and to push through new innovations through a research-led approach. Rafiq also believes workers outside of the CGT industry, but with skills transferable to it, could be a worthwhile resource to tap. “It’s also about repurposing people from the sector, such as those people already working in therapeutic areas, or other sectors, such as supply chain, manufacturing, engineering and logistics,” he says. “It’s a very inter-disciplinary field led by clinicians and scientists, but to be sustainable we need to address the cost of manufacturing with the help of engineers and biochemical engineers, and it may require data scientists and project managers to help. We are seeing different expertise coming into the field and we’re now starting to see the impact of overall deliverables. It’s a team endeavour rather than a solo pursuit. It’s very exciting.” As for Rafiq’s prediction about the future of CGT, he estimates that, in around five to seven years, we will see a huge change in how diseases are treated with these new and emerging therapies. While clearly not an overnight result, this will happen, at least, once innovations feed through and help to drive down the costs of treatments to the thousands, rather than the hundreds of thousands they are currently at. ●
World Pharmaceutical Frontiers /
www.worldpharmaceuticals.net
Designua/
Shutterstock.com
Page 1 |
Page 2 |
Page 3 |
Page 4 |
Page 5 |
Page 6 |
Page 7 |
Page 8 |
Page 9 |
Page 10 |
Page 11 |
Page 12 |
Page 13 |
Page 14 |
Page 15 |
Page 16 |
Page 17 |
Page 18 |
Page 19 |
Page 20 |
Page 21 |
Page 22 |
Page 23 |
Page 24 |
Page 25 |
Page 26 |
Page 27 |
Page 28 |
Page 29 |
Page 30 |
Page 31 |
Page 32 |
Page 33 |
Page 34 |
Page 35 |
Page 36 |
Page 37 |
Page 38 |
Page 39 |
Page 40 |
Page 41 |
Page 42 |
Page 43 |
Page 44 |
Page 45 |
Page 46 |
Page 47 |
Page 48 |
Page 49 |
Page 50 |
Page 51 |
Page 52 |
Page 53
