Scientific breakthroughs, such as the development of CRISPR gene editing technology, have ushered in a new era of medicine. One particularly promising field is regenerative medicine, which uses cell therapies and gene therapies to repair or even replace human cells, genes and tissues in order to restore the normal function of the body.
We spoke with Dr. Malin Parmar, Professor of Cellular Neuroscience at Lund University in Sweden, and Dr. Nicole Paulk, Professor of AAV Gene Therapy at the University of California, San Francisco, about recent developments- and future opportunities. As pioneers in their respective areas of research and development, they explained what is needed to take cell and gene therapies mainstream and shared with us why they are optimistic about the future of these cutting-edge technologies.
What is the status quo in cell and gene therapy?
Malin Parmar: There are various ways that stem cells have been used to treat diseases or injuries. Hematopoietic stem cells for example, so the blood stem cells that give rise to other blood cells, have been used to restore a healthy blood system in people living with leukaemia. Stem cells have also been used to grow skin grafts for patients who have suffered from severe burns. These are just two examples of what’s currently possible.
Nicole Paulk: In gene therapy, we have started seeing promising leaps during the past five to ten years. Currently, there are two FDA-approved gene therapies to treat two rare diseases, spinal muscular atrophy and inherited retinal dystrophy. At the same time, there are hundreds of gene therapy trials in the clinic, mostly for monogenic disorders, so diseases that are caused by variations in a single gene.
What is it about cell therapies and gene therapies that has led experts such as yourselves to believe that they are taking medicine to new places?
Nicole Paulk: When I started my PhD about a decade and a half ago, we were using viruses as tools to perform genetic medicine for the first time. It was still in a concept stage and we had not really done this effectively in the clinic, yet.
Fast forward 10 to 15 years, we can now give patients, who live with life-threatening genetic disorders, a functional copy of the affected gene that they need to survive. These patients are with us today because of that. To me, this is just mind-blowing.
I see gene therapy as the third big transformational moment in medicine: the first moment was the invention of antibiotics to treat bacterial infections, the second was the discovery of vaccines, and the third is the development of gene therapy. My mind boggles at the fact that we can give kids, who had no hope, the chance to lead a normal life. They’ll meet all their normal growth milestones. They’ll be playing soccer with their friends.
Malin Parmar: I think that the future is bright, and I think that we will be able to prevent and reverse diseases or at least treat them much better than we do today. And this is both in the fields of gene therapy and cell therapy. Along the way, we will need to learn more about cells. We need to learn more about genes.
Ultimately, we will be able to combine cell therapy approaches and gene editing to make healthy versions of our own cells and create patient-specific treatments. These therapies promise to be very effective in treating tough diseases like cancer.
What needs to happen to turn this future vision into reality?
Nicole Paulk: We try to develop technologies that allow us to produce gene therapies faster, easier, and thereby more cost-effective, so that we can distribute them equitably throughout the entire world. The ambition is that no matter where you're born, if you need a gene therapy, you have access to it.
Malin Parmar: I think one of the major struggles is a bit of a race against time. Not only does it take a long time to develop and manufacture cell therapies, which in turn affects the cost of development and the cost of the approved drug. It also takes time until you see the full therapeutic effect.
I really wish that this field would move faster. But we have a saying in Sweden that you need to rush slowly, because the risk is that if you rush too fast, you will fail along the way and you won't get to your end goal. It’s a real challenge to rush slowly in this field.
I feel with the patients who are desperately waiting for these new therapies. Sitting with my mom, whom I lost to ALS, all I could do is wish that the field would go faster. But as a scientist I know we have to move at the pace that the science allows. It is a challenge to rush slowly.
Cell and Gene Therapy at Bayer
Commentary by Jost Reinhard, Head of Cell & Gene Therapy, Bayer Pharmaceuticals
If we as a pharmaceutical company are serious about our purpose ‘Science for a better life’, there is no way around cell and gene therapy.
With cell and gene therapies, we are moving beyond symptomatic treatments with the potential to reverse disease. There are different perceptions when it comes to cell and gene therapies – an emotional perception of the magic when seeing the potential results for patients, and a great fascination with science. Personally, I perceive cell and gene therapies as a long journey. For one thing because it’s complicated and for another - more importantly - because we are entering a field marked by a lot of innovation, which will come stepwise.
Together with our partners AskBio, Atara Biotherapeutics, BlueRock, and Mammoth Biosciences we are investing in know-how and infrastructure to bring safe and effective cell and gene therapies to patients globally. We live by our vision of ‘Health for All’ and are truly committed to making these breakthrough therapies a reality for patients around the world.
Based on what Bayer, the industry and the broader healthcare ecosystem have already achieved, I think the future of these cutting-edge technologies is enormously exciting. I am convinced that cell and gene therapies will create lasting improvements for patients whose medical needs are not yet met by today’s treatment options.