Following her appearance on this week’s The Naked Scientists podcast on 'Drug Discovery', Dr Magda Papadaki, ABPI’s Head of Manufacturing Innovation, writes about realising the potential of cell and gene therapy.
In a momentous decision last month, the US Food and Drug Administration has approved the use of Kymriah, propelling the practice of medicine – and particularly the treatment of cancer - into a new era.
Approved for the treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukaemia (ALL) that is refractory or in second or later relapse, Novartis' Kymriah shows an unprecedented 83 percent overall remission rate, and becomes the first ever CAR-T cell therapy to win approval in the US. For children and young adults, a particularly sensitive patient population, it offers a fundamentally new treatment approach.
Currently, paediatric and young adult patients with B-cell acute lymphoblastic leukaemia have extremely limited treatment options and historically poor outcomes. To date they have to undergo multiple rounds of treatment with chemotherapy, radiation, targeted therapy or stem cell transplant, yet less than 10 percent survive for more than five years.
CAR-T offers a new treatment approach in that it is specifically produced for each individual patient, making manufacturing more complicated than for any other type of medicine. The immune system T cells are taken from a patient's blood and their DNA is reprogrammed in the lab to express targeting mechanisms called chimeric antigen receptors (CARs). The altered cells, which are now instructed to hunt the patient's cancer cells, are infused back into the same patient, a process that currently takes about two weeks. Delivering this procedure cost-effectively and in a way that it can meet demand remains a persisting challenge.
Although CAR-T cell therapy has shown the greatest promise against blood cancers, there is strong hope that their potential can go beyond one type of cancer, meaning Kymriah could pave the way for a new standard of care in a variety of intractable, relapsed or refractory cancers.
Novartis picked up rights to Kymriah under an agreement with the University of Pennsylvania in 2012, highlighting the "urgent need" to continue building strong partnerships between academic innovators and the powerhouses of global biopharmaceutical developers. As we continue to shed new light to those hardest to treat cancers and optimize the growing wave of promising CAR-T therapies, collaborations such as these will be increasingly important in turning scientific breakthroughs into unprecedented patient outcomes.
Building the UK global foundations
The FDA's decision is also great news for UK biopharma Oxford BioMedica, which is Novartis' manufacturer for the lentiviral vector – an essential tool for delivering the edited gene into the cell. With more than 50 cell and gene therapy developers in the UK, Oxford Biomedica is just one of the potential success stories in this space that marks significant new opportunities for growth and productivity in Britain
Yet despite CAR-T therapy's immense promise, there are many anticipated challenges ahead that need to be worked through. Realising the potential of CAR-T needs a joined up system that encourages investment in innovation, supports research and development, provides clear regulatory guidelines and education, a positive uptake environment, and stimulates a new generation of skilled workers. This is at the heart of the UK Advanced Therapy Manufacturing Task Force, which is a nationwide effort to 'anchor' the development, manufacturing and use of CAR-T's and similar advanced therapies in the UK to secure the country's position as a world class hub.
Investing now to build and grow the UK's scientific and operational base, as set out by the MMIP and the ABPI, could enable local firms to claim bigger share of the global market and further position the UK to be the home of developing and delivering the very latest medical breakthroughs.
That's why the new Life Sciences Industrial Strategy, and its funding mechanisms such as the Industrial Strategy Challenge Fund, couldn't come at a better time. The Strategy can be the vehicle for the UK to unite behind and build the necessary conditions to develop, manufacture and adopt transformative new therapies such as CAR-T treatments, fast-forwarding Britain into what's set to be the next golden era of medicine.