Advanced therapy platforms have the potential to fight cancer in novel ways


Unlike conventional medicines, advanced therapy platforms treat diseases in unique ways, such as precisely targeting cancers, modulating the immune system or targeting the fundamental cause of genetic diseases. The treatments address the underlying mechanisms of the disease, rather than just managing symptoms. These platforms have the potential to bring innovative treatments to patients.

With one of the largest research and development programmes in the industry, Novartis is making significant investments in this area, specifically AAV-based gene therapy, CAR-T cell therapy, radioligand therapy (RLT) and CRISPR-based therapy. Of these, CAR-T cell therapy and RLT have already shown promise in treating certain cancers and are being explored in others.

“Novartis recognises that advanced therapy platforms have the potential to reimagine medicine. Their transformative power is only just beginning to be understood as our understanding of cell biology and genetic engineering advances. With the right systems in place they have the potential to deliver promising results for the NHS in the future,” says Mari Scheiffele, general manager, UK and Ireland, at Novartis Oncology.

Promising treatments

CAR-T cell therapy, or chimeric antigen receptor T-cell therapy, involves genetically modifying a patient’s own immune cells to recognise and destroy cancer cells. This personalised treatment can help some patients whose disease has resisted all other forms of medication.

Research has also shown that CAR-T cells remain in the body and continue to be active for long periods of time. Therefore, unlike many other cancer drugs, CAR-T cell therapy is designed to be a one-time treatment, helping the individual continue to fight the disease throughout their life.

“While current medicines have enabled substantial improvements in cancer patient outcomes over recent years, in many cancer types there’s still a need for novel treatment options. CAR-T cell therapy has the potential to offer long-term remission and improve quality of life. Novartis was the first company to offer a licensed CAR-T product in the UK and is investigating multiple cancer types for this platform,” says Ed Jenkins, franchise head of Cell and Gene Therapy at Novartis UK.

RLT is also growing in importance for cancer care. This form of nuclear medicine delivers radiation directly to cancer cells via the bloodstream, leaving healthy cells largely unaffected. Targeted isotopes cause DNA strands to break, disrupting the cells’ ability to replicate and subsequently trigger cell death. The use of RLT has expanded significantly in recent years.

Ecosystem approach needed

Key in the delivery of these advanced therapy platforms is collaboration. Unlike conventional therapies, these platforms often require specific clinical expertise and unique facilities for administration. They can also require specialised manufacturing processes and a strong skills base. Certain treatments are manufactured individually for each patient. In the example of CAR-T cell therapy, the hospital becomes a key player in the manufacturing process as it is responsible for collecting the patient’s T-cells and sending them to Novartis.

These advanced therapy platforms often need to be delivered within specific time limitations. This became a challenge with the outbreak of the coronavirus pandemic. Novartis’ CAR-T cell therapy is manufactured outside the UK and the flight restrictions that were put in place could potentially have caused delays in delivering this treatment to patients. Working with the government and regulatory bodies, Novartis was able to identify alternative routes to bring this important treatment to patients.

In the case of RLT, due to the nature of the medicine, patients who receive this treatment must do so in isolation rooms. Many of these rooms were repurposed to prioritise patients with COVID-19. Working with the NHS and the private hospital networks, Advanced Accelerator Applications (AAA), the Novartis-affiliated company that makes RLT, was able to ensure cancer patients could still receive their treatment.

We’re keen to continue working with the NHS to increase capacity to offer our advances in cancer therapies, so every eligible patient in the UK can benefit

“We cannot deliver these highly complex and innovative treatments alone. We need an ecosystem approach. That’s why we are collaborating with UK government officials, Health Technology Assessment bodies, the NHS, hospital networks, academia, doctors, commissioners, industry partners, as well as patients and patient groups, to support improvements in diagnostics and therapeutics,” says Jeevan Virk, general manager of AAA UK, Ireland, Nordics and Baltics.

Looking ahead

In the last decade, there has been a tremendous effort by researchers, clinicians, scientists and doctors in academic and industry settings to develop this class of therapies, which are now resulting in real outcomes. For instance, the number of patients to be treated with advanced therapy platforms is predicted to rise to 10,000 every year over the next decade, from roughly 200 patients in 2018.

Industry is keen to target major unmet needs in a wide array of cancers using these innovative therapies and the NHS is looking to play a leading role in the development of cutting-edge technology in a new era of personalised medicine.

The UK has the potential to be a leader in the development, discovery and adoption of advanced therapy platforms. There is an opportunity to work with industry partners to fast-track promising treatments into clinical trials, develop integrated data systems and harness real-world evidence to identify areas that are of greatest patient benefit.

“Advanced therapy platforms have the potential to extend and increase the quality of life. We’re keen to continue working with the NHS to increase capacity to offer our advances in cancer therapies, so every eligible patient in the UK can benefit,” Scheiffele concludes.

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Q&A: Radioligand therapy has a promising future for cancer treatment

Q&A Jeevan Virk, Advanced Accelerator Applications, UK and Ireland General Manager

Jeevan Virk, Advanced Accelerator Applications, UK and Ireland General Manager

Radiopharmaceuticals are increasingly used for diagnosing cancer, as well as treating the disease.  For one company, born at CERN, targeted radioligand therapies are showing promise in cancer management. Jeevan Virk, Advanced Accelerator Applications, UK and Ireland General Manager, talks about this exciting advanced therapy platform

What is targeted radioligand therapy?

Radioligand therapy (RLT) and radioligand imaging involve a precision medicine approach combining a radioisotope or radionuclide with a targeting molecule or “ligand” that binds to specific markers or receptors on cancer cells. Both the radioisotope and the ligand can be changed, depending on the  specific type of cancer a patient has and whether it’s being used for diagnosis or therapy.

For diagnosis, a radionuclide emitting a type of energy that’s detected with special cameras can be used to visualise the location of the cancer using a PET-CT scan. These scans can also identify the right patients for treatment.

When used as a treatment, a radionuclide that emits therapeutic radiation can be used with the same ligand. After infusion into the bloodstream, RLT targets the cancer cells, releasing radiation and causing breaks in the cancer cells’ DNA and cell damage. This either kills the cancer cells or prevents them from replicating. The most common form of therapeutic radiation used (beta) only travels a few millimetres, which minimises damage to surrounding healthy tissues.

What can this type of nuclear medicine achieve? 

You normally associate radiotherapy or chemotherapy with patients feeling sick while they receive treatment. RLT, in contrast, is well tolerated. For people with cancer, this therapy can provide them with an option that can slow the progression of their disease for a significant period of time, allowing patients to go about their everyday lives.

How are you embracing new technology?

We’re using data and digital processes to drive RLT, whether it’s the screening of new radioactive isotopes or new targeting molecules, monitoring patients’ responses or informing them about the innovative therapies they’re taking. Every therapeutic treatment is personalised, so data is very much part of this process. Shortening supply chain timeframes and production schedules requires careful data management to ensure patients receive their treatment on time.

What are the challenges with deploying these therapies?

We activate the radioisotope in a nuclear reactor in the Netherlands. It’s then shipped to RLT manufacturing sites, where the isotope is combined with the targeting molecule through specialised chemical synthesis. From there, it’s sent to hospitals around the world for delivery to patients within 72 hours, otherwise the radioactivity degrades to a level where the dose isn’t effective.

It’s a logistical challenge, yet we were successful, even during the height of the COVID-19 pandemic, in delivering medicines to patients every day around the globe, despite travel restrictions. This puts us in good stead for the end of the Brexit transition period on December 31. We’ve been working with the Department of Health medical supplies team to make sure our treatments are not disrupted because of the UK leaving the European Union.

Is the UK a good place to develop this type of cancer therapy?

The UK and the NHS have taken a leadership position in clinical trials research for RLT. We hope patients here will have access to these cutting-edge treatments far earlier than other nations. An ecosystem approach thrives in the UK, bringing patients, patient advocacy groups, researchers and universities, as well as industry and the NHS together. COVID-19 has also shown how important patient outcomes are.

What does the future hold for RLT?

The possibilities for this advanced therapy platform in cancer are exciting and in some ways we are at the start of this journey. At the moment, it’s only being used to tackle rare forms of cancer, but we want to use this form of nuclear medicine to treat more common cancers, bringing targeted RLT to many more patients.

This is why we are researching new isotopes with different energy levels, as well as different targeting molecules that can target different kinds of solid tumours. The amount of basic research that is happening each year is remarkable, with an exponential growth in new publications. It means our understanding is gathering momentum too. These are exciting times. Watch this space.

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