Dr Paul Stoffels: The 'human biology' revolution

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The industry and medical research community have the potential to deliver transformational change for patients based on the recent dramatic increase in human biology, according to Dr Paul Stoffels, Johnson & Johnson vice chairman and chief scientific officer.

Dr Stoffels, who sits on the company’s global leadership team and reports directly to CEO Alex Gorsky, is currently in Australia to meet with researchers, partners and key decision-makers including health minister Greg Hunt.

“We have very good relationships in Australia,” he said. “We have partnerships with universities and academic centres. The biotech community is also quite strong. 

“We have around 50 collaborations in Australia and many are now in the clinical stage. I am here to support the facilitation of our collaborations and it is very good to have Australia as a partner for the global advancement of medicine.

“The science can come from anywhere and that includes Australia. This is why we focus on maintaining good relationships across the board.”

He told BiotechDispatch medical research for every disease has been greatly enhanced by the knowledge of why a patient will respond to individual treatments.

“The whole sequencing and diagnostic effort. A lot of things have become very much clearer. We also have more modalities in terms of the tools we have available to work with.

“We started with small molecules and vaccines but now have the multi-targeted antibodies, cell therapy, gene therapy and proteins. We have these modalities that enable intervention in the body.

“Small molecules were the basis for many revolutionary medicines over 50 or 60 years. We went from the early discovery of drugs for tuberculosis, antibiotics, antihypertensive drugs - these were all small molecules.

“There are many more opportunities in small molecules but we then went to antibodies and, it took 20 years, but they opened the door to a range of new possibilities for patients. Today, inflammation diseases, psoriasis, IBD, Crohn's disease, ulcerative colitis and many more inflammatory diseases can be managed with antibodies.

“Then you get to the multi-specific antibodies where you can target two or three things at the same time. So again, it took 10 years to get there, but you are seeing exceptional proof of concepts that mean you can control blood cancers. This is a proof of concept that can be recreated with different types of modalities.

“Then gene therapy with CRISPR. You can now see the first types but it will become mainstream.

“This biology combined with the platforms provides so much potential but we cannot forget about diagnostics. We can more effectively identify the disease and then target that disease.”

Dr Stoffels says the expansion in knowledge regarding human biology has had a dramatic impact on the conduct of medical research globally.

“Now a lot of the work is not all done in the pharmaceutical industry. It is done in a combination of the pharmaceutical biotech environment or academic centres.

“Translational research is typically a 15-year process. It is rarely something that is just around the corner. This means you have different types of organisations and ‘innovation networks’ involved in translation."

He said Johnson & Johnson is focussed on new technologies but also the application of small molecules in developing countries.

“We work on the most advanced technologies for developed countries but, when we think about their application more broadly, we have to think about modalities that are more accessible, simple and safe to use.

“This is why the new human biology is so important because it will help us get to better small molecules and vaccines. We are working on HIV and Ebola vaccines because, if you want to have an impact on a global scale, you have to make it simple.

“You cannot get to billions of patients if you make a complex modality. An example of transformational modality is in mental health and HIV with long-acting injectables, where you can go from one pill once a day to one injection every second month.

“As a global company, we have to remain focused on the healthcare problems of the world and that often means thinking about the most applicable, simple and accessible modalities.”

Dr Stoffels said the company’s R&D model is based on a matrix. “We focus on the disease, find the science and use the right modality.”

“We have several centres of excellence where we have capability organised by campus. We have oncologists using vaccines to prime tumours. We have experts in devices and our consumer business who use digital and different material science.”

Johnson & Johnson Innovation maintains a significant organisational presence in Australia led by Kathy Connell.

“Australia is geographically far away and that is why we have people on the ground. We have around 2,000 people in Australia and that means a lot of capability. We also have the innovation network.”

Dr Stoffells said the company currently maintains over 1,000 early-stage collaborations and 500 companies in incubators globally.

“That is a big funnel to later-stage products but it is a very significant logistical effort. How do you keep track of all that? It is a global organisation with people like Kathy who are really the connective tissue.

“We always try to keep a window into Johnson & Johnson. We know through Kathy and our network here what is happening in Australia so we do not need 500 people to fly in. We have a local office with local experts. They connect to the global organisation and alert us to the very good things in science happening here.

“Of course, not everything will come to fruition. We fail more than we succeed in everything we do in pharmaceuticals and medical devices, but that is that experience you build over the years and what helps you make the right choices.”

Dr Stoffels said the future will see patients screened annually with one simple blood test for cancer. “Many tumours will be taken out or cured early. Personalised medicine will dominate with every patient guided by diagnostics for therapy. We will be preventing more disease because there will be earlier intervention. Importantly, we will also get the science to more people in the world, regardless of where they live.”