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How Countries Are Regulating Gene Therapy: Ethical, Medical, and Cost Issues

Europe may be lagging behind in clinical trials of GMOs but this may show its positive sides as the medical and ethical evaluation of the consequences of such advanced medical research is still in its infancy. Currently, Europe is far behind the other developed continents in gene therapy clinical trials. How does the regulatory framework affect the successful development of genetic engineering and what other issues must countries consider?

It All Started With Dolly

On the 5th of July 1996, the “world’s most famous sheep” was born, not in a sheep pen or paddock but in a science research laboratory. Dolly, a female sheep, was cloned by researchers at the Roseline Institute, part of the University of Edinburgh including Keith Campbell, and Ian Wilmut in collaboration with PPL Therapeutics, a biotech company. An adult somatic cell from a mammary gland was used for the production using a process known as somatic cell nuclear transfer.

Major Genetic Engineering Concerns

The cloning of Dolly raised major concerns in not just gene cloning but in genetic engineering generally. The attention of the world and the media was drawn and government regulatory measures heightened even for gene therapy. Gene therapy and genetic enhancement concerns were not anymore seen as mere science-fiction. 

The next year, a regulatory body was convened in the UK to address the practice and in a July 2018 ruling, the European Court of Justice decreed that gene editing is by law genetic modification and must be regulated as such.

However, the government in many countries have continued to fund and encourage gene therapy for genetic disorders like progeria, a disease malfunction that makes children age much faster than normal, and dementia, a genetic disorder that impairs the ability to remember, think, or make decisions that interfere with doing everyday activities and many others. But such support is yet to be garnered for gene enhancement “designer baby”.

Gene Therapy Regulations in the European Union

The first attempt to treat diseases with the help of gene therapy was in 1990, making 4-year-old Ashanti DeSilva the first recipient, and the EU home to foremost gene therapy medical practices. But lately, they may be lagging behind in the area of advanced and modern clinical trials. 

Is Europe Far Behind Other Developed Continents?

Due to their strict legislation applying to Genetically Modified Organisms (GMO), Europe is far behind other developed continents in clinical trials of Advanced Therapy Medicinal Products (ATMPs). Initially, the GMO regulations were made with agricultural products in mind, but the authorization procedure for clinical trials involving ATMPs requires extra layers of regulation to comply with.

When compared to other continents, North America and Asia have recorded significant increases in the number of clinical trials between 2014-2018. Europe has remained unchanged. North America increased by 36% while Asia increased by 28%, and the EU by <2% on a global scale of ATMPs clinical trials which increased by 32%

Due to their strict legislation applying to Genetically Modified Organisms (GMO), Europe is far behind other developed continents in clinical trials of Advanced Therapy Medicinal Products (ATMPs).

Clinical trials initiated between 2014-2018 according to continents.

Source: Alliance for Regenerative Medicine

Decisions on the regulation of GMOs are interpreted differently according to competent organizations of the EU member nations, giving rise to different outcomes. GMO and clinical trial legislation are legally valid but there is no defining modus operandi for how they should interoperate.

ATMPs available in Europe are only 11. This shows the rigors and the demanding standards placed by European Medicines Agency (EMA), the regulatory arm for gene therapies, on the research institutions. 

Cost Challenges Confronting Clinical Research, Approval and Administration

A great deal of financial investment is required to maintain the efficacy, quality, safety, and transparency standards set by the body. To the medical research institutions, meeting these standards is the most challenging aspect of clinical research for gene therapies. With the imposition of new regulations, it even becomes more difficult for the industry. In 2007, when new regulations were set, clinical research institutions found it very hard to cope.

Should We Expect New Gene Therapies Coming Soon?

Due to the tough regulations, time, and financial restraints, we may begin to expect new gene therapies in the coming years although about 500 clinical trials have been carried out in the last decade using the ATMPs. This was also the case with the gene therapies available in the market today. They were approved after long years of waiting for market authorization.

Strimvelis gained approval in 2016 after two decades becoming the first ex-vivo stem cell gene therapy used in treating patients with Severe Combined Immunodeficiency caused by Adenosine Deaminase deficiency (ADA-SCID), a very rare disease.

Chondrocelect, a treatment for single symptomatic cartilage defects of the femoral condyle of the knee in adults was approved in 2009 after nine years of waiting. But just last year, the company filed for their market authorization to be withdrawn from Europe due to unsustainable costs of not getting enough patients to pay for the treatment. This is yet another challenge confronting the industry.

Expenses Connected With Developing Gene Therapies

The development of gene therapy is very expensive. The treatments are priced to the tune of hundreds of thousands of euros and private insurance companies hesitate to pay for the expenses. In most cases, the occurrence of the disease in Europe is very minimal. The company then finds it difficult to make profits after decades of investment in the research and awaiting approval. A case in point is Glybera.

Glybera was used for the treatment of a very rare hereditary disease called lipoprotein lipase deficiency (LPLD). The drug development took decades and even after approval in 2012, it took the company three more years to get a patient willing to pay the throat-cutting sum of about 1 million euros. The drug was only used to treat Elisabeth Steinhagen-Thiessen. The remaining three doses were administered to patients for one euro each. In October 2017, the company filed for withdrawal from the European market. 

EMA holds the right to approve and withdraw market authorization across the continent but various nations have their competent authorities that make regulations as well. 

Ethical Concerns In Gene Therapy and Enhancements

Gene therapy for intervention in the human genome for aesthetic enhancements in children is a new field that has come under discussion. In this practice, a particular gene is modified according to an individual’s desire. This is purely a non-medical purpose and can either use somatic cell genetic engineering or gem cell genetic engineering to modify traits like color of the eye and hair, height, educational skills, etc.

Somatic therapies target genes in specific types of cells like lung cells, skin cells, blood cells while germline modifications when applied to embryos, sperm, or eggs, alter the genes in all the baby’s cells. Somatic cell modifications are non-inheritable, affecting only the treated individual. 

Until now, market authorization of this form of gene therapy has not been given in the EU and the U.S. This may be due to the technical, ethical, and moral issues that remain to be resolved.

Major Challenge Facing Regulatory Institutions In Terms Of Gene Therapies

The major challenge facing regulatory institutions remains to make a distinction between gene therapies for treating diseases and for enhancements. Would a gene enhancement for slimness be considered an aesthetic purpose for an obese person? What about permanent gene modification for bald hair?

Other ethical concerns plaguing the regulation of the sector are: would there be a social divide between those who have been genetically enhanced and those who have not? Will it spur the market for preferred physical traits?

Many countries placed an outright ban on the genetic enhancement of embryos while some others have placed a ban on regulations. In 2015, the first gene editing carried out on an embryo took place in China.

Diagram showing country regulations of gene editing in embryos in a research carried out by Motoko Araki and Tetsuya Ishii of Hokkaido University in Japan in 2014

Diagram showing country regulations of gene editing in embryos in a research carried out by Motoko Araki and Tetsuya Ishii of Hokkaido University in Japan in 2014. Source: Business Insider.

While groundbreaking research is being carried out in this field, regulators should take into consideration whether genetic enhancement will hamper the principle of human dignity, justice, and the responsibility to pass on a healthy and unadulterated life to future generations. What would the future generation think of the present?


Veronica Ugwu
Veronica Ugwu
Veronica Ugwu is a writer for RegTech Global, with her enthusiasm for tech and business.


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