Next generation cell and gene therapies: fine tuning the promise


31-10-2019
Image: Miltenyi Biotec ClinMACS Prodigy machine, GSK R&D Cell and Gene Therapy, Stevenage Photographer: Richard Moran, May 2018

On 19 November, the UK BioBeat19 summit goes to Stevenage to discuss the potential of cell and gene therapy and how to accelerate these transformational medicines.

The organisers from GSK (Victoria Higgins) and BioBeat (Miranda Weston-Smith) spoke to two panellists who gave a sneak peek of their remarks and agree wholeheartedly that the discovery side and clinical side work best when they are teamed up.

Sophie Papa, an oncologist at Guy’s Cancer at Guy’s and St Thomas’ NHS Trust, and Aisha Hasan, a clinical development lead at GSK, both recognise the big challenge ahead for cell therapy researchers: to dial up efficacy and dial down toxicity.

Cell and gene therapies, with their remarkable potential to transform medicine, have seen some important but hard-won milestones: it took 20 years of combined academic and industry research to deliver the first gene therapy approval in 2016 and today there are two CAR-Ts approved for haematological malignancies. Whilst CAR-Ts recognise proteins expressed on the tumour cell surface, making them ideal for targeting blood cancers, more complicated — but with greater potential to address solid tumours — are the gene modified TCR-T technologies. These harness the power of T cells to specifically target and destroy tumours – even on the inside of cells. TCR-Ts come with an additional level of complexity, but potentially open the door to a range of untreatable cancer types.

Looking at the TCR opportunity is where Sophie Papa sees the inherent trade-off between risk and benefit as an academic clinician who’s now evaluating modified T-cell based therapies in clinical trials. Sophie urges her peers to take courage.  “It is important to be brave and tolerant of certain toxicities. Academic clinicians and drug researchers need to work closely together to engage the regulators in early discussion, so that we can move cell therapies earlier in treatment schedules as soon as feasible. Timing is critical to enable patients to be treated when they are physically fit so they can better tolerate these complex and potentially toxic treatments.”

From her perspective, this is not an ‘either/or,’ but an area where discussion and open dialogue will allow us to make the most of the opportunity.   “By allowing clinical academics to play a lead role in developing guidelines to manage patient safety, we can address legitimate concerns but not let them stand in the way of clinical development.”

Aisha brings the perspective of drug discovery and development and starts by asking what is in the realm of the possible from a design perspective. “A superior T-cell therapy will require engineering approaches that enhance efficacy on one-end while also incorporating switches to minimize toxicity.”

For example, in a counter-intuitive way, a T-cell with high-killing capacity actually can create dangerous levels of inflammation in the body, due to the rapid death of cancer cells. But the beauty of drug design opens up options:  “By building a switch within the engineered T-cells, researchers can inactivate the T-cells and prevent harm to the patient.”

But this creative problem solving requires open dialogue between clinicians and pharma.  Aisha says:  “The more we talk about clinical need and toxicity benchmarks, the more sophisticated we can be when developing the next generation of enhanced engineered cell therapies.”

There’s no doubt that the challenges of delivering cell and gene therapy span the full spectrum of issues related to medicine development. However, the potential for both curative therapy and commercial opportunity is tremendous. The scientific, clinical, technical, regulatory and commercial challenges are all surmountable when everyone in the ecosystem work together towards a shared goal, united by an unwavering focus on the patient.

Sophie and Aisha are speaking about the translational journey from science to bedside at the BioBeat19 summit.

Sophie Papa is a Clinical Reader and Honorary Consultant Medical Oncologist, Guy’s Cancer at Guy’s and St Thomas’ NHS Trust. Aisha Hasan is the Clinical Development Lead for Cell and Gene Therapy Oncology at GSK.

Image: Miltenyi Biotec ClinMACS Prodigy machine, GSK R&D Cell and Gene Therapy, Stevenage
Photographer: Richard Moran, May 2018

BioBeat19 summit

Sophie and Aisha spoke with Victoria Higgins, Senior Director of the Cambridge-GSK Alliance and Miranda Weston-Smith, Founder of BioBeat.  Victoria and Miranda are organising the BioBeat19 summit in conjunction with Sally Ann Forsyth, CEO of Stevenage Bioscience Catalyst.   

The BioBeat19 summit on Accelerating cell and gene therapy takes place from 1-6pm on Tuesday 19 November at GSK Stevenage.

The cell and gene therapy sector is expanding fast, bringing undreamt of opportunities for scientists, entrepreneurs, innovators and investors.  

The BioBeat19 summit will explore the journey from innovative science to patients and what value means for different people and organisations.  We’re honoured to be joined by a stellar international line-up of women speakers drawn from the commercial, public and academic sectors.  Emma Walmsley, CEO, GSK will deliver the keynote address and the event is open to all.

The summit will take place in the GSK Auditorium, GSK Research and Development Campus, Stevenage SG1 2NY (Satnav SG1 2FX), 1.00-6.00 pm on Tuesday 19 November 2019.

Guarantee your place by registering today at www.biobeat19.org>>>

To read more information, click here.

Miranda helps early stage biomedical businesses to attract investment and develop their business strategy. Miranda founded and runs BioBeat, a programme to inspire the next wave of bioentrepreneurs and business leaders

Miranda Weston-Smith