Cell and Gene Therapy, (CGT) has the potential to transform 21st-century medicine. These innovative approaches offer new avenues for treating and potentially curing a wide range of diseases and conditions by targeting the underlying causes at the genetic and cellular levels.
Cell therapy involves the transplantation or manipulation of cells to restore or enhance their function in the body. This approach holds great promise for conditions such as cancer, neurological disorders, and autoimmune diseases. Ever since Emily Whitehead became the first patient to be cured using CAR-T cell therapy, which involves modifying a patient's immune cells to recognize and attack cancer cells, there have been 500+ CAR-T cell therapies in various clinical trials and 4 approved treatments.
Gene therapy, on the other hand, aims to treat disease by modifying or replacing a patient's genes. It involves introducing genetic material into a person's cells to correct a faulty gene or provide new instructions to the cells. Gene therapy has already demonstrated noteworthy progress in clinical trials treating genetic disorders like spinal muscular atrophy and inherited retinal diseases.
On June 22, 2023, the FDA approved Elevidys, the first of its kind gene therapy for the treatment of Duchenne Muscular Dystrophy, (DMD), in pediatric patients 4-5 years of age. They used recombinant gene therapy to deliver a gene that codes for a ‘micro-dystrophin’ protein. This is accomplished using an adeno-associated virus to deliver the genetic payload to the cells of the body. This first of its kind therapy will treat the underlying cause of the disease rather than slowing its progression. Elevidys was approved through the Accelerated Approval pathway to address the unmet medical need of a life-threatening disease. With a narrow target of approved patients, ambulatory 4–5-year-olds, and a shortened form of the protein, Elevidys is not perfect, first attempts at addressing complex diseases rarely are, but it is an important first step to proving the ability to address a systemic genetic disease by correcting the underlying defect.
The potential of cell and gene therapy lies in their ability to use the body’s own immune system to attack cancer or correct the underlying genetic defect, and these new capabilities raise the curtain on a whole new field of medicine. As our understanding of genetics and cellular biology deepens, we can expect advancements in these fields to lead to more targeted and effective therapies.
However, it is important to note that despite the promising results and ongoing research, there are still challenges to overcome before cell and gene therapy becomes widespread in medical practice. New and complex therapies always come with a big price tag. CAR-T cell therapies average $350,000 – 450,000 and Elevidys carries a price tag of $3.2 million, but these costs need to be balanced against the cumulative costs of traditional treatments and therapies. We must ask ourselves does it make more sense to address a disease with a new, highly effective therapy as opposed to older, cheaper, less effective therapies that are highly burdensome for the patient, their families, and society as a whole?
If we added up all the cost of traditional treatments and long-term support and compared them to the cost of new therapies, we might just find that it makes more sense, not only morally but financially, to treat patients with newer and more expensive therapies. Make these options the first choice rather than the last resort.
Overall, while cell and gene therapy hold immense potential, their transformative impact on 21st-century medicine will depend on continued scientific advancements, regulatory support, and the ability to address the challenges associated with their cost and implementation.