Cell and gene therapies can radically change treatment

Mariecar Jara-Puyod, Senior Reporter

Cell and gene therapies combined with the burgeoning digital health and artificial intelligence shall remain to be a nucleus and continually pivot the intensely-competitive pharmaceutical industry, accelerating solutions for improved health and better outcomes.

This was emphasised at the “Virtual Pharma Media Day” of the healthcare-agriculture global enterprise Bayer on Wednesday (noon to 2.30 pm, UAE time), attended by 200 journalists from 30 countries. Cell therapy is the delivery or injection of “functioning cells into a patient’s body to achieve a medicinal effect-to prevent, manage or cure a certain illness or to repair and regenerate damaged cells.”

Gene therapy uses a genetic material into a “patient’s cell so that cells which have been lacking the correct instructions to work properly receive the genetic information that will allow them to restore their function.” The increasing significance of cell and gene therapies, as well as digital health and artificial intelligence, relative to pervading health challenges such as the yet-to-resolve central nervous system degenerative disorder Parkinson’s disease, was well-discussed with a panel of experts.

At the open forum, Bayer AG Board of Management member/Bayer Pharmaceuticals Division president Stefan Oelrich, at some point, commented: “Medicine is an accessory to care.” His statement drove the female science journalist-moderator to quip: “That statement is revolutionary.” According to Oelrich, “the biomedical and technical revolution that is transforming healthcare at an unprecedented pace is taking place right now. Our goal is to bring breakthrough treatments to patients and make healthcare systems more sustainable in the mid-and-long-term.” On his take on whether cell and gene therapies would drive down healthcare costs considering all the consultations, diagnostics, hospitalisation, treatment and post-treatments that go with it and at times are out-of-pockets, Oelrich said: “We need to radically re-think how the cost of medicine (is associated with) the total cost of care like (relative to the taxpayers).”

The reason for cell and gene therapies, including digital health and artificial intelligence to be taken seriously and acted upon astutely; such as acquisitions of or partnerships with individuals in these areas of interest.

In the “Digital Health” session, Philippe University of Marburg (Germany) Artificial Intelligence in Medicine professor Dr. Martin Hirsch pointed out that trust is a “fragile word” and so the digitalisation of healthcare must be comprehensive yet understandable and transparent for patients and other users. One Drop founder/CEO Jeff Dachis, a diabetic, said he established the digital health platform due to his frustration and disappointment over the traditional mode of physician-patient interactions. He believes that a well-conceptualised app and digital platform substantiates the delivery of integrated healthcare needs.

In the “Cell & Gene Therapy: The Future of Cellular Medicine” session, BlueRock Therapeutics (US-Canada-based engineered cell therapy company founder/chief executive officer (CEO) Emile Nuwaysir and Asklepios BioPharmaceuticals (US-headquartered Genome Medicine/adeno-associated virus gene therapy company) co-founder/CEO Sheila Mikhail pointed out why these matter. Mikhail said over 7,000 diseases may arise from one malfunctioning gene. She added that a global study had revealed that 20 per cent of the world population (1.6 billion of the 7.8 billion) have at least one deformed gene. Nuwaysir and Mikhail explained that as genetic diseases are caused by faulty genes and so chemical/traditional medicines cannot cure these and thus treatment is limited, “cell and gene therapies have the potential to shift the paradigm of the disease treatment to cure with potentially encouraging effects for patients’ lives. Nuwaysir’s and Mikhail’ companies are now subsidiaries of Bayer AG as the drug firm believes in their undertakings.

Incidentally, BlueRock Therapeutics, as approved by the US Food and Drug Administration, is going to begin within the first quarter of 2021 the Phase 1 clinical trial the injection of pluripotent stem-cell derived dopaminergic neurons in patients suffering from Parkinson’s disease.