Trend: It's a miracle

A trend indicates a direction of change in values and needs which is driven by forces and manifests itself already in various ways within certain groups in society.

Health is challenged by the emergence of new infectious diseases that are spreading in our increasingly connected world and by diseases that are resistant to antibiotics. Breakthrough treatments are helping to counteract some of these problems and will be needed for the future. The COVID-19 pandemic built on and drove innovation in the area of vaccines. mRNA vaccine technology has been successful and is hugely promising for the future. We are finding new medicines and repurposing older medicines (such as Remdesivir), that are being successfully used to fight coronaviruses and other diseases.

The use of revolutionary technologies such as the genetic scissors ‘Crispr’ and cell-based therapies are on the rise. New technologies for treating heart failure could replace cardiac transplantation. As knowledge about complex diseases such as Cancer and AIDS increases, researchers are finding increasingly precise methods of treatment. Targeted therapies, based on a variety of drugs that target specific molecular pathways within individuals (identified through screening), and new technologies (e.g. nanomedicines targeting specific tissues), allow physicians to identify and treat the patients that are likely to respond to specific ‘personalised’ treatments. 

This Trend is part of the Megatrend  Shifting health challenges

Manifestations

Developments happening in certain groups in society that indicate examples of change related to the trend.

Cell and gene-based treatments

Advanced therapy medicinal products (ATMPs) are medicines based on knowledge about genes, tissues or cells. They offer groundbreaking opportunities for the treatment of disease and injury. Gene therapy uses genes to treat a disorder by inserting a gene into a patient's cells instead of using drugs or surgery. A mutated gene that causes disease can be replaced with a functional copy, for e.g. in immunotherapy techniques, cancers, viral diseases or inherited disorders. Cell-based therapy involves modifying a patient’s own cells or using cells from a donor to fight disease and alleviate conditions in organs (such as the eye, heart, skeletal muscle or skin).

Many clinical uses of stem cells are under investigation. Stem cells are like ‘baby cells’, and can grow and develop to become any ‘adult’ cell of the human body. Stem cell therapy can repair diseased or injured tissue. Doctors routinely and increasingly use stem cells that come from bone marrow or blood in transplant procedures to treat patients with cancer and disorders of the blood and immune system. The World Economic Forum has postulated that stem cells could be the medical innovation of this century, and note strong growth in the cancer stem-cell market and the rapid increase in start-up companies. Ethics and research integrity will be critical for the future concerning embryonic stem cells and unregulated clinics, as well as human enhancement and inequalities.

Signals of change: European Medicines Agency, ISSCR, EC

CRISPR gene editing

CRISPR-Cas9 is the so-called ‘genetic scissors’. It is a revolutionary gene editing technology that allows for precise changes to genes. It is widely used to modify DNA in cells in research labs and research is opening up the possibility of achieving therapeutic genome editing in humans. While cloning is not new (i.e. cutting and pasting DNA sequences), the CRISPR editing technique is easier, cheaper, faster and precise. It is based on a bacterial antiviral defence system and can cut a cell's genome at a specific location, allowing a piece of DNA to be removed and/or a new piece to be added. It has possibilities for new medicines across a broad range of diseases. For e.g. in the treatment of inherited genetic diseases, cancer, regenerative medicine and rare diseases. It has just had its first breakthrough success and has been delivered as a medicine successfully in a human body directly. Gene editing can also be used to eradicate diseases transmitted by animals, or mosquitoes.

The rapid development of CRISPR technology and its applications - including its potential to modify the human germline - have opened up massive interest, as well as ethical and regulatory debate. There are diverging views and more knowledge about the potential risks to the individual, society and future generations is needed. While the technology is advancing fast, despite its precision - it is known that there are often unintended DNA changes too. Also, reaching challenging areas within the human body (brain, heart and muscles) is still out of reach and cost is a big concern: treating sickle cell disease with CRISPR therapy costs about $2 million (EUR 1.71 million) per patient. 

Signals of change: BBC future, WHO, NPR, Fortune

New age of vaccinology

Vaccines have transformed health and save millions of lives annually. Vaccines exploit the ability of the immune system to respond to (and remember) pathogens such as viruses that might cause an infectious disease. Transparency and clear communication about vaccines has never been more important, with public hesitation and misinformation about the effectiveness and safety of vaccines and new vaccines circulating, and ongoing COVID-19-induced lockdowns.

mRNA vaccines are a new type of vaccine. To trigger an immune response, many vaccines consist of a weakened or inactivated pathogen (germ), or a small piece of it, often delivered by deactivated viruses. mRNA vaccines are different. They teach cells how to make a protein from that same pathogen, and its the protein that triggers the immune response. That immune response produces antibodies and they, (along with other immune responses), protect against infection and disease.

The potential of using our body’s system in this way to treat illness in the future goes far beyond the current pandemic. mRNA vaccines were under investigation for cancer, influenza, Zika virus, rabies and cytomegalovirus. The COVID-19 mRNA vaccines are a breakthrough that has built on this earlier work. Future mRNA vaccine technology might allow one vaccine to provide protection against multiple diseases more successfully and thus reduce the number of vaccine injections needed. Yet despite tremendous progress and potential, far too many people around the world still have insufficient access to vaccines.

Signals of change: WHO, Fortune, AAAS Science

Interesting questions

What might this trend imply, what should we be aware of, what could we study in more depth? Some ideas:

• What if there is no long term customer service for specialised health products (bionic eye)?

• Where do we draw the line between human and robot?

• How will future generations be affected by genome editing?

• How can we ensure responsible use of radical gene editing techniques in embryos?

• What if the ethical and social aspects don't get attention they deserve?

• Cell and gene-based therapies may only be afford by the rich. Will inequalities become broader? 

• Will healthcare providers be able to provide the most suitable medicine, at the right dose, for the right person, for everyone including sick children at the right time, at a reasonable cost? And if so, by when, 2040, 2050? Ever?

• How do we find and ensure a reasonable balance between investment, profit, and shared benefit for the citizen and for the patient?

• With mRNA vaccines for cancer and other diseases as well as infectious agents, and modern personalised treatments and new medicines, will sickness become a thing of the past?