Cell & Gene Therapy is a growing area of possibility for saving and improving the lives of people with blood cancers, blood disorders. In this blog, we speak to Dr Diana Hernandez about her work and what the future holds.
Please tell us a little bit about yourself
My name is Diana Hernandez, and I am head of translational immunotherapy at the Anthony Nolan Research Institute, and I've been working here just over five years. I am a geneticist as my first degree, and then I did a PhD in the genetics of cancer at the University of Birmingham a long time ago. I worked in academia for about 15 years, then worked in biotech for 6 years before coming to work at Anthony Nolan. So I've been working in science for all my life, I guess.
Why science? Why research?
My father had a pharmaceutical company back in Colombia where I'm from, and I was always interested in, you know, mixing things in laboratories. I thought test tubes were really cool, so then I started kind of playing with the idea of what kind of careers could I go in to.
I watched a documentary when I was about 12 by Dame Kay Davies, who is a very famous British geneticist talking about the discovery of the gene for muscular dystrophy. And I thought; that's it. That's what I want to do.
I found a way of coming to the UK to do genetics and stayed and never wanted to leave the profession.
What is Cell & Gene Therapy?
People think this is a brand-new thing – and in part, it is, but it’s more of an evolution of sorts. Traditionally we treat diseases by giving people chemicals of some description in the form of medicines, but with cell therapy we are instead given cells to correct what is wrong in the body. Haematopoietic cell transplants are effectively a form of cell therapy and one of the earliest ones – we’ve been doing it for about 50 or so years now. It involves taking cells from a healthy donor and putting them into a patient whose immune system is no longer working properly for whatever reason, effectively replacing that system. It’s called “cell therapy” as we’re using cells as vehicles to cure or treat a person, but it is also a regenerative therapy, as we expect the cells to stay in the body and continue working for the lifetime of the patient.
There are other forms of cell therapy that do not involve regeneration, cells are transiently infused into a patient, and after a while they die off. New advanced therapies such as CAR-T therapy have taken things to the next level by ‘arming’ the transplanted cells with genes capable of targeting and eliminating tumour (cancer) cells. They are placed in patient’s body to go on to do what they do best, which is killing these abnormal cells, but they are only transiently there.
Gene therapy was first trialled quite a while ago, but the first few attempts were not super successful – this unfortunately held the industry back quite a bit at the time. But significant improvements have certainly been made! The type we used now, called ‘ex vivo gene therapy,’ is used to treat patients born with blood disorders such as Wiskott-Aldrich syndrome, for example, which is what Anthony Nolan himself had. It involves removing a small number of blood cells from a patient’s body and then manipulating them in the lab. We basically correct the defective genes they carry in their cells and reintroduce these cells into the patient’s body, repopulating their immune system with cells which function normally.
What are the main objectives in this line of research right now?
We want to refine these therapies even more. When chemotherapy was discovered, it was ground-breaking. We had discovered a chemical that could kill actively dividing cells - and cancer cells divide much more quickly than regular cells. But we’re all aware of the side effects – there’s a lot of collateral damage with chemotherapy. What we’re trying do now is work out how we can use cells to target the cause of disease and either eliminate it (in the case of cancer) or correct it (in the case of genetic disorders) with minimal collateral damage. That way we can make this therapy less horrible to put up with by eliminating a lot of the side effects. It’s about continuing to refine and create ‘precision medicines’ as we call them.
There is also a desire to make some of these therapies cheaper. The advantage of chemotherapy is that it’s very cheap and very effective, and the majority of people respond quite well to it. But for Cell & Gene therapy it’s about getting it to a point where it can be mass produced (rather than being so “handmade” and “bespoke” as it is currently), so the costs can be reduced, and more people can have access to it. That’s the dream.
What kind of difference has this research made, or could it make, for patients?
When advanced therapies were first rolled out, they were given to patients in palliative care. Most of these people had already been treated with chemotherapy so this was their only option. Even if only half of them were saved with treatment, it’s still giving them a second chance of life when there was nothing else left. Even if it only gives them an extra 5 years, that’s so incredibly valuable for those people and their families. I think any new treatment that allows people to have a fairly good quality extension of their life is pretty life changing. In the future we hope these therapies will make truly curative treatments a reality.
What do you enjoy most about this work?
The fact that you have new things every day – you never know what’s going to happen. There’s often times a lot of failure to get one success, but it’s always new and I love that.
And the people; I love mentoring new researchers. I love the sparkle in their eye when something is working, and they’ve found something really interesting. I think that’s what’s truly amazing for me.