Gene therapy has become a technological golden snitch: the end of an era for many genetic disorders if we can grasp the treatment’s potential. However, the technology comes with a steep cost, creating a barrier to entry for many families and preventing researchers from investigating the full range of treatment possibilities. As originally reported in Pulmonary Hypertension News, the high price tag has sparked a debate over who is supposed to pay for these therapies.
Former commissioner of the FDA, Scott Gottlieb, stresses that gene therapy needs be made affordable for those who need it. He says we need insurance companies and public health systems to budget in these life-changing therapies that enable patients to entirely avoid otherwise dire situations. Right now, insurance companies often exclude gene therapies in their policies because of fears that it will balloon costs. However,for many patients, these therapies means life or death. According to Pulmonary Hypertension News, Gottleib said, “it’s imperative that all patients have access [to these new therapies], regardless of someone’s ability to afford them.”
However, if the treatments are not subsidized, the treatments could be extremely costly, meaning only families that could pay get access while less privileged patients would have even fewer affordable treatments because there would be less investment in less effective, cheaper methods. The only options would be extremely expensive, and currently, insurance companies are not including gene therapies in their policies. For many patients, these therapies are a need, not an option.
Who Does Gene Therapy Help?
Gene therapy has the potential to address birth defects that about 3% of American children are born with. They very often take the lives of these children early on. While right now it is targeted towards specific illnesses, these are far from the only diseases that could tap into this developing resource. Types of vision or hearing loss and fertility issues have therapies in the works; about 80% of rare diseases are caused by genetics, and thus often benefit from developments in gene therapy.
What is Gene Therapy?
Gene therapies are unique from traditional medications because the therapy alters the individuals genetic makeup. The procedure directly inserts functional genes into individuals who lack the gene or have an extra or defective one. The new genes replace whatever function may be lacking. Usually, people with these genetic disorders have very few resources and hope of survival is minimal. Most people with these disorders pass away in childhood. (http://www.biolyse.ca/gene-therapy-pros-and-cons/) The novel approach of gene therapy prevents the disease from developing in the first place and could potential do away with these genetic diseases entirely. Furthermore, the faulty gene is removed from the bloodline so that it is no longer passed on to the next generation. This is the first chance of a cure for many incurable diseases.
Why Gene Therapy?
At the National Organization for Rare Disorders (NORD) Summit 2019, Mark Rothera, president and CEO of gene therapy company Orchard Therapeutics, said that after decades of experience in rare disease pharmaceutical creation, he had switched over to gene therapy because of how incredible the findings had been. It totally revolutionized rare disease treatment. He saw how this one time treatment could last a lifetime versus the typical life-long care required for these diseases. This is very distinct from previous therapies which are often a permanent fixture in patients lives, with constant check ins, medications, and interventions. If you have one of these diseases, it may seem like a no-brainer.
The Gene Therapy That Saved Two of Her Children’s Lives
Amy Price described her experience of discovering that three of her children had metachromic leukodystrophy (MLD). Over time, this rare progressive disease causes loss of movement and intellect as well as seizures, difficulty talking, and personality changes. Because it is progressive, eventually patients lose the ability to walk, talk, see, and function entirely.
Her first child received the diagnosis at age two. They identified the disease after it had advanced to a point at which she could not be saved. However, the experienced motivated them to test their second child, only months old at the time. This time, the disease had not yet progressed to the point of no return. They caught the disease in time for him to receive the life-saving gene therapy. He received genes that corrected his faulty glial cells, enabling them to produce the missing enzyme; this stopped the disease before it even began. The same procedure was done for the third child born with the disease. Both of them live symptom-free.
In The End…
Gene therapies are enormously impactful and exciting, but require investment and subsidizing to reach their great potential and be accessible for those in need. Because this tool is tremendously powerful, it can be dangerous. Anything that gives us access into the fundamental mechanics of humanity gives us the ability to do both great good and bad with it. However, altering a gene that is destructive, crippling, and life-ending is a very different question than altering healthy genes for the sake of it. This is about utilizing a technology to save lives that would otherwise have their time cut short.
