Gene and Cell Therapies: Shaping the National Strategy

Gene and Cell Therapies: Shaping the National Strategy

The recently published National Strategy for Gene and Cell-Based Therapies is intended to link the players in this innovative field of medicine throughout Germany and to speed up the introduction of treatment approaches into clinical care. Four clinical experts from Hamburg were involved in the development of the strategy. What is most important to them?

What are gene and cell therapies?

Gene and cell therapies (CGT) are among the most important innovations in biomedical research and modern healthcare. They have the potential to fundamentally change the way cancer, autoimmune diseases, neurodegenerative diseases and many rare genetic diseases are treated.

Living medicines

The new therapies are based on genes, tissues or cells and therefore often contain living components. The products can be better tailored to individual patients than traditional drugs. Since gene and cell therapies act directly on the genetic cause of a disease process, they are particularly suitable for the treatment of diseases that have previously been difficult or impossible to treat.

In the EU, they are legally classified as advanced therapy medicinal products (ATMPs). The European Medicines Agency (EMA) is responsible for their approval.

Healing with cells

Put simply, cell therapy means treating diseases with living cells: modified and cultivated cells are transferred to the patient to repair or replace damaged cells. Or cells are administered that have been modified to expand their function.

First-generation cell therapies are already improving the lives of patients around the world: blood stem cell transplants are a routine procedure in hospitals. The use of advanced technologies is enabling the development of therapies for blood diseases, including some types of leukemia and lymphoma. In fact, there are already several approved cell therapies for cancer that are currently being used to treat patients.

The CAR-T cell therapies are particularly groundbreaking: highly specialized immune cells, the T cells, are taken from the patient. These are equipped with an artificial molecule, the chimeric antigen receptor, or CAR for short, in the laboratory. Equipped with the receptor, the CAR-T cells can target cancer cells or other cells in the patient and fight them effectively. Tissue replacement cultivated in the laboratory is also a form of cell-based therapy.

Targeting the genes

In gene therapy, genetic information in the form of DNA or RNA is used for treatment. This can mean that genetic material is introduced into, removed from or modified in the cells of a patient in order to treat an inherited or acquired disease.

Several gene therapies have been approved for the treatment of various diseases, including some types of eye disease, spinal muscular atrophy and beta thalassemia, a blood disorder. GCTs are already an integral part of clinical medicine. Many researchers and clinicians are convinced that they will herald a new era of therapy.

What are the objectives of the National Strategy for Gene and Cell Therapies?

Although hundreds of clinical trials are underway to develop gene and cell therapeutics, only a few products have been approved in Europe so far. There is a great need for research to improve efficiency, safety and availability. While basic research and application-oriented technology development for GCT are already being successfully carried out in Germany, the translation, i.e. the transfer of promising research approaches from research and development to patient care, remains a particular challenge. Germany is in danger of losing its international lead in this area of key medical technologies.

In order to regain a leading role in this area, actors from science, business, politics and society must work together more closely. This is precisely the aim of the National Strategy for Gene and Cell-Based Therapies, which was presented on June 12 at the Berlin Futurium and handed over to Federal Research Minister Bettina Stark-Watzinger.

Around 150 experts from various stakeholder groups have developed the 140-page paper (PDF Download) and a roadmap for improving patient care and strengthening Germany’s position in the field of gene and cell-based therapies. The Berlin Institute of Health at the Charité (BIH) moderated and coordinated this multi-stakeholder process. The federal government will provide 44 million euros for the design and implementation of the strategy’s measures between 2023 and 2026, with a further 4 million coming from the state of Berlin.

The national initiative aims to promote and develop the future-oriented medical field of GCTs in all areas in Germany and identifies measures. The aim is to improve the framework conditions for the development of therapies in research, production and application. This is to be achieved on the one hand through political advice and clear and better legal regulations, but also through closer networking between universities, research centers and industry. The training of specialists and the transfer of technology are to be strongly supported.

The picture shows three people, with Francis Ayuk from the University Medical Center Hamburg-Eppendorf in the center of the picture. © Svea Pietschmann
Francis Ayuk from the University Medical Center Hamburg-Eppendorf (center of picture) was among the major contributors to the National Strategy for Gene and Cell Therapies. © Svea Pietschmann

What does the strategy mean for the Life Science Nord cluster?

The University Medical Center Hamburg-Eppendorf (UKE) is the leading center for research and the application of gene and cell therapies in northern Germany. No fewer than four experts from the UKE were involved in the creation of the National Strategy for GCT.

The UKE’s Stem Cell Transplantation Clinic is the largest stem cell transplant center in Germany, with around 200 allogeneic blood stem cell transplants a year, and one of the largest in Europe. Professor Francis Ayuk, Deputy Director of the Clinic, was involved as a spokesperson for the Market Authorization and Transition to Care working group.

“Working on the strategy in a multi-stakeholder format was new to me and an excellent experience,” says Ayuk. Thousands of patients are already benefiting from GCT in various fields of medicine, and many new GCT therapies are currently entering the clinic. For the senior physician, all measures that ensure high-quality, safe and efficient treatment for patients have priority.

“But we also need sustainable translation processes. This involves reducing bureaucratic hurdles and ensuring adequate remuneration through flexible reimbursement and care models. If we can get this complex issue of care right, we will have achieved a great deal.” His UKE colleague Katja Weisel, Professor and Deputy Director of the Center for Oncology and University Cancer Center Hamburg (UCCH), was also involved in the working group.

Boris Fehse is a professor of cell and gene therapy at the UKE’s stem cell transplantation clinic. His research includes the development of safe methods for the genetic modification of blood cells for use in gene therapy and cancer research. Together with organoid researcher Madeleine Bunders, Fehse was involved in the “Research and Development” working group of the National CGT Strategy.

Leading CAR-T cell therapy center in Germany

“The UKE is one of the largest CAR-T cell therapy centers in Germany,” says Fehse. Almost 200 patients have already been treated with CAR-Ts. So far, the genetically modified immune cells have been successfully used to treat various malignant diseases of the blood and lymphatic system.

“At the UKE, patients with multiple sclerosis were recently treated with CAR-T cells for the first time in the world. These cells attack B cells, which play a key role in the disease,” says Fehse.

Fehse is convinced that the national strategy will raise awareness of the growing importance of GCTs among all stakeholders, especially in politics, the authorities and among funding agencies. “We expect better financial support for both the development of therapies and the necessary clinical trials. In addition, we hope that the now clearly defined coordination channels will help to reduce bureaucracy and thus accelerate research into new therapies.”

The northern German location should also benefit greatly from this. Fehse’s team holds a number of patents in the field of GCT and, together with the UKE team, would like to bring a gene therapy for the treatment of HIV into clinical development.

Text: Philipp Graf

More on this topic in the LSN Magazine
Stem Cell Medicine in the North: The Report

Hero picture: Contributing experts surround German Federal Minister of Education and Research Bettina Stark-Watzinger, who is presenting the printed version of the National Gene and Cell Therapy Strategy. © Svea Pietschmann

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