What makes ATMPs special?
In the context of the INTEGRATE project, four very different diseases are addressed, all of which share one common feature: With the help of ATMPs (Advanced Therapy Medicinal Products), disease progression can be halted, an outbreak can be prevented, or remission (disease regression) can be achieved. The unique aspect of ATMPs is that they can treat diseases in a completely new way by directly targeting the underlying cause. ATMPs include gene therapies, cell therapies, and tissue replacement therapies, which differ significantly from conventional medications. Some important areas of application include:
- Cancer Treatment
ATMPs, particularly chimeric antigen receptor (CAR) T-cell therapies, are used to treat certain blood cancers, among others B-cell lymphomas and leukemias. By genetically modifying patient’s T-cells, cancer cells are specifically targeted and destroyed. This offers a novel approach to treat previously particularly difficult to treat cancers. - Neurological Diseases
For diseases such as spinal muscular atrophy (SMA), where motor neurons degenerate, a developed gene therapy can correct the genetic defect underlying the disease. Especially when applied early, improvements in muscle strength and control in affected patients can be achieved. - Metabolic and Genetic Disorders
Metachromatic leukodystrophy (MLD), a rare genetic disorder that leads to progressive neurological degeneration, can be treated via/using gene therapy. This treatment offers the possibility of halting disease progression by replacing the defective gene responsible for the disorder. - Inherited Immunodeficiencies
Adenosine deaminase deficiency (ADA-SCID) is a rare, genetically inherited immune deficiency, where a lack of the enzyme adenosine deaminase (ADA) disrupts the development of immune cells. Affected patients are vulnerable to severe infections. The available gene therapy corrects the underlying genetic defect. ATMPs also offer promising possibilities for the treatment of a variety of other genetically inherited diseases by addressing the underlying genetic causes and providing long-term, if not permanent, therapeutic solutions. Gene replacement and genome modification are two important mechanisms used to treat these diseases.
In summary: ATMPs offer personalized, targeted treatments that address the root causes of diseases and have the potential to mediate for permanent cures. These characteristics make ATMPs a particularly promising and forward-looking class of therapies.
Challenges in the Use of ATMPS
ATMP-Therapies for the four diseases addressed in INTEGRATE-ATMP have been used in Germany for only a few years. Since these therapies involve genetic modifications high-quality standards have been defined for ATMPs and their application. This comes with several challenges:
- Existing healthcare structures in outpatient care and follow-up must be adapted (structural and financial adjustments to accommodate the increased workload).
- Treatment outcomes needs to be systematically recorded to obtain extensive information on the effectiveness and safety of the therapies.
- The subjective perception of patients, including health-related quality of life and satisfaction during the course of treatment, needs to be assessed.
ATMP Quality Assurance Guidelines
The guidelines regulate the requirements for the quality of application of Advanced Therapy Medicinal Products to ensure high-quality patient care and the proper application of ATMPs in the interest of patient safety.
Link: ATMP Quality Assurance Guidelines issued by the Gemeinsamer Bundesausschuss (G-BA, Federal Joint Committee) - ATMP-Qualitätssicherungs-Richtlinie - Gemeinsamer Bundesausschuss