Jacques Galipeau, MD
Associate Dean for Therapeutics Development at the University of Wisconsin-Madison and President of the International Society for Cell & Gene Therapy (ISCT)
Jaap Jan Boelens, MD, PhD
Chief of the Pediatric Stem Cell Transplantation and Cellular Therapies Service at Memorial Sloan Kettering Cancer Center and North America Regional Vice-President Elect for the International Society for Cell & Gene Therapy (ISCT)
In recent years, cell and gene therapies have experienced tremendous growth with an increasing number of products receiving regulatory approvals.
Cell and gene therapies — which encompass cell transplants — and genetic modifications and gene-editing of cells represent a significant paradigm shift in the treatment of rare diseases due to their unprecedented effectiveness and curative potential.
Cell and gene therapies for rare diseases
Cell and gene therapy technologies already exist as FDA or EMA-approved cures of uncommon and rare diseases such as metachromatic leukodystrophy, ADA-SCID (Severe Combined Immune Deficiency), cerebral adrenoleukodystrophy and transfusion-dependent thalassemia major.
One example, allogeneic bone marrow transplantation (allo BMT) — a type of cell therapy that involves replacing diseased marrow with healthy marrow from a donor — has already been proven to be a curative treatment option for rare monogenetic bone marrow diseases such as primary immune deficiencies, haemoglobinopathies and lysosomal storage diseases (LSD).
However, as a result of using donor cells, allo BMT carries a risk of treatment-related toxicities, infections, residual disease (in cases of LSDs) and mortality which limits its use to the most severe cases. In contrast, autologous gene therapies involve gene addition or gene-editing of patients’ own cells offering a potentially safer and more effective solution.
Autologous gene therapies involve gene addition or gene-editing of patients’ own cells offering a potentially safer and more effective solution.
Jaap Jan Boelens, MD, PhD
Therapy opportunities for rare and ultra-rare diseases
These novel therapies can also be used to treat patients with milder cases who have historically not been considered for allogeneic bone marrow transplantation because of the associated risk of toxicity and mortality.
These same gene engineering tools can be readily repurposed to address the needs of other rare and ultra-rare diseases. The opportunity for the medical field is in defining novel means of clinical development and sustainable patient deployment for these therapies that meet risk-adapted regulatory requirements and improve upon the current standard of care.
Enabling and amplifying progress in the field
We emphasise that advancing clinical development and deployment will require collaboration between stakeholders working at all stages of development. To enable this growth, we highlight the key role of the International Society for Cell & Gene Therapy (ISCT),a collaborative global community for professionals to communicate and learn from one another to avoid pitfalls and improve success rates of translating scientific discovery.
Through strong relationships with regulatory agencies, academic institutions and industry partners, ISCT drives the translation of research into safe, effective and accessible treatments for patients with rare diseases globally.
ISCT is a global expert-guided community of cell and gene therapy professionals bridging scientific research, regulatory requirements and commercial interests to improve lives through safe and effective cell and gene therapies.
