This following opinion editorial was originally published by the Vancouver Sun.
Opinion: It’s the first time that a therapy using CRISPR has been approved for the treatment of a condition in humans. What does that mean for the future of medical science?
A new gene therapy, utilizing the gene-editing tool known as CRISPR to treat sickle-cell disease, has received approval in the U.K., the U.S., and the EU. This milestone marks a potential breakthrough in alleviating the severe and life-threatening symptoms of sickle-cell disease while respecting ethical boundaries.
That’s not the only milestone that has been reached; it is also the first time that a therapy using CRISPR has been approved for the treatment of a condition in humans.
CRISPR, an acronym for Clustered Regularly Interspaced Short Palindromic Repeats, has revolutionized genetic editing within existing organisms.
When the gene-editing tool first rose to public prominence in 2015, it came with controversy. Its groundbreaking potential captured the public’s interest but was initially met with a mix of enthusiasm and skepticism. Developed by doctors Jennifer Doudna, Emmanuelle Charpentier, Feng Zhang and Francisco Mojica, CRISPR’s journey has been one of significant evolution since its inception.
However, in 2018, the scientific community faced a critical ethical breach when scientist He Jiankui edited embryos, purportedly conferring resistance to HIV, which resulted in the birth of twins in China. This incident solidified the consensus that CRISPR should not be employed for editing in sperm, eggs or embryos, reinforcing ethical boundaries surrounding its use. Amid this controversy, the recent decisions by the U.K., U.S. and EU to approve CRISPR for therapeutic use are particularly noteworthy.
A potential cure for sickle cell disease
Sickle-cell disease is among the most common genetically inherited conditions in Canada. Sickle Cell Disease Canada estimates that 6,000 Canadians are living with the disease, which causes debilitating symptoms due to a single genetic mutation.
In an ongoing trial, Vertex Pharmaceuticals and CRISPR Therapeutics treated 30 patients with sickle-cell anemia, witnessing a remarkable transition from multiple pain incidents to zero incidents in just one year. Similarly, 39 out of 42 patients receiving similar treatment for beta thalassemia no longer required blood transfusions to counter severe anemia.
Beyond the technological stride forward, the success of this trial holds particular significance for marginalized populations — including individuals of African, Indian and Middle Eastern descent — that pharmaceutical companies and the health care industry have historically overlooked. These advancements offer new-found hope for Canadians grappling with this condition.
However, the likelihood of CRISPR gene therapy gaining approval in Canada following its acceptance in the U.K., U.S. and EU remains uncertain. The approval process in Canada may require clinical trials with Canadian patients and could span from six months to two years. Beyond approval, the implementation of CRISPR treatments in Canada hinges on decisions made by federal or provincial health care systems and considerations of cost, as the treatment carries a hefty price tag.
Remaining challenges
Despite this recent progress, uncertainties prevail concerning the treatment’s long-term effects, preventing doctors from labelling it as a definitive cure. Cost and resource limitations pose significant challenges, with the price of the treatment in the U.S. being set at $2.2 million per patient or more.
On the medical front, obstacles such as immunogenicity, where the immune system targets and fights back against the engineered cells, remain significant hurdles.
From a business perspective, navigating CRISPR licensing agreements, predominantly held by a limited number of companies, poses hurdles for market accessibility and may discourage new entrants into the field.
While challenges persist, transitioning from laboratory experiments to human treatments within a decade represents a remarkable achievement in medical science.
The future of CRISPR
Now that CRISPR gene therapy has gained a toehold as an approved medical treatment, is there a path ahead for expanding its applications to other genetic conditions? These include diseases of the blood, eye and liver that are easiest to target due to their accessibility by gene editing delivery.
CRISPR gene therapy’s historic approval unlocks doors for addressing previously untreatable genetic disorders and advancing personalized medicine. By replacing genes in vivo — inside living organisms — CRISPR could revolutionize treatment approaches. The FDA’s establishment of a new pathway for treating rare diseases underscores CRISPR’s versatility and potential beyond singular applications.
To ensure CRISPR’s widespread success, overcoming challenges in effectively delivering CRISPR to target tissues beyond the liver, eyes and blood is paramount. Additionally, addressing the high cost of treatment and broadening market accessibility are essential for maximizing CRISPR’s positive impact on patients’ lives.
This moment of CRISPR’s approval for treating sickle-cell disease marks a significant milestone in medical science and may kick off a new era in personalized medicine. It paves the way for transformative innovations in genetic medicine and offers hope to patients with previously untreatable genetic disorders.
