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Advanced Therapies Week Superplenary Round-up: Patient Advocacy at the Heart of Advanced Therapies

Anna Osborne
2 February 2022
For this round-up, Anna Osborne summarises an insightful – and at times emotional – Superplenary session, that stood to remind the industry why we do what we do and the importance of events such as Advanced Therapies Week for cell and gene therapy development.

After what has been an unprecedented two years, the Phacilitate team has been ecstatic to welcome back our in-person Advanced Therapies Week in Miami.

Industry experts joined to watch Phacilitate Executive Vice President, Kim Barnes, as she kicked off the conference’s  Superplenary on Tuesday 25th January 2022.

As with all aspects of life, the global pandemic brought its challenges to the advanced therapies industry, yet it is clear that the last two years have been a successful time of growth and accomplishment and there is plenty to celebrate; we have seen a 58% increase in clinical trial activity, with 847 mergers and acquisitions taking place last year.

Superplenary host Rob Allen – General Manager and Senior Principle at Dark Horse Consulting – notes that despite positive outcomes,  there are many challenges the industry still faces, some of which have been intensified by Covid-19. Many clinical trials had to be put on hold or slowed down due to the functional difficulties a pandemic presented. Before welcoming his guests, Rob comment that to move forward we must have an “appetite to overcome the challenges we need to beat”.

As we enter a week of bringing together the brightest minds and innovators in the global advanced therapies industry to build partnerships and accelerate the progression of advanced therapeutics, systems and technologies, it is important to reflect on the reasons why these therapies are so vital.

Initially joining Rob on stage were Lisa Ward and Tom Whitehead, two parents whose children were instrumental in the first clinical stages of advanced therapeutics for ALL and DIPG, respectively. In touching segments, Tom and Lisa shared their stories and the impact advancing these therapies had for their children Emily and Jace, as well as describing their bravery in taking part in the initial stage trials, and their continued work in raising awareness and facilitating advanced therapy treatments for other patients in similar circumstances.

Tom has been heading up The Emily Whitehead Foundation for the last seven years, celebrating the foundation’s seventh birthday this year as well as the 10 year anniversary since Emily became the  first patient to receive CAR-T cell therapy to successfully treat her cancer. He poignantly comments how there “was no hope for [ALL patients] and now there is”. Emily was given just a 1 in 1000 chance of waking up from a coma, and after 23 days on experimental CAR-T treatment, she was cancer free. Last week she took her driving test. Next year she will be going to college. It is these real life stories which reinforce the gravity Advanced Therapeutics have in saving lives.

Lisa Ward’s son, Jace, faced a 6–9 month life expectancy following his extremely rare DIPG diagnosis. There were no options for treatment. Staring this in the face, Jace showed tremendous courage, campaigning for DIPG foundation – TOUGH2GETHER, with his mum. Hearing of an investigational drug for high-grade gliomas – ONC201 – in its early stages of human clinical trials, Jace took on the risk of wading into the unknown. His tumour showed a significant decrease in size and extended his life, though sadly Jace passed away last year. His legacy lives on, not only through the impact his story has in motivating industry leaders to continue to accelerate these therapies, but also through the work of his mother and the children who have since been able to gain access to ONC201.

Both Tom and Lisa are vocal to praise the work of the industry experts, however without patients like Emily and Jace, the progression of these innovations from a conception to commercialisation reaching patients to save lives would not be possible. The advanced industry is a world relying on communication and collaboration for success. Patient advocacy for these therapeutics can be underestimated. As Lisa summarises: patients, parents, innovators and investors have to be “brave enough to move forward and walk into areas that feel risky”. Not one area within advanced therapies development is alone in this risk. All parties involved are paramount in moving the industry forward.

Introduced on stage next was Shepherd Mpofu, Chief Medical Officer at Novartis Gene Therapies. Shepherd emphasised Novartis’ focus on the end-to-end journey of a product from clinical to commercial environments, keeping the patient experience at the forefront at all times. Engaging with the patients not only serves as a touching reminder of the underlying purpose for these therapies, but also allows for companies to iron out patient-specific challenges immediately.

Shepherd acknowledged the generic challenges facing advanced therapy commercialisation, including manufacturing costs. However, Novartis is keen to view expenses in terms of therapeutic value and the lifesaving potential of developing specific drugs. The focus for Novartis – as well as other biotechs and GMPs alike – is to create a robust, scalable and sustainable efficient process. Creating flexible solutions allows these therapies to reach more patients, including those in low and middle income countries.

Finally joining the panel was Craig Shepherd, Senior Managing Director of Blackstone Life Sciences which focuses its $4.6 billion fund to invest in programs at late Phase III clinical stages to facilitate therapeutics’ progression to commercialisation. Craig referred to three pillars Blackstone considers when investing in companies and products, which are: efficacy, safety and capability. The funding they provide is with the precedent of enabling a smooth commercial transition with lower costs and in a quicker time frame, in order to get these therapies to patients faster.

In a comment that mimics the sentiment of the panel, Craig reflected on the compelling last five years that have seen advanced therapies have an immense impact on patients, offering so much hope to so many. As Lisa mentioned in an earlier moving reference to advanced therapies for DIPG treatment, if you “save a child, you save a family”.

As we look ahead to what the cell and gene therapy space will look like in future years and the gaps we need to approach, it is clear to see how the collaboration between patients, biotechs and investors to address key challenges will enable the industry to facilitate earlier intervention. Supporting technological innovation, allocating more resources and an updated expense model for novel therapies will all build upon the success and pace we are currently witnessing within advanced therapies.

It is clear that advanced therapies represent the next pillar of medicine, and if there is one thing I took away with me from the Suplerplenary fire-side chats, it’s that the future is looking bright.

Join the network to watch the Superplenary on demand >>>