Do patients understand the risk of reshaping a genome?
- Gene editing is already changing the landscape of cancer treatment, but concerns remain that patients don't adequately understand the dangers inherent to the cutting and pasting of DNA.
- Informed consent — the concept that patients know the risks of an investigational medicine before they sign up to take it — has become a hot-button issue in the gene therapy space, according to a recent forum hosted by the Harvard T.H. Chan School of Public Health and NBC News Digital.
- Historically, a drug moved into clinical trials based on the verdict of a review board and the level of informed consent from participants, but that doesn't work with gene editing, said George Annas, director of Boston University's Center for Health Law, Ethics & Human Rights, during the forum. "Whenever you go into a human population for the first time … the major issue is safety and risk. Is this the right thing to do?"
Kayla Richardson was less than two years old when she received an infusion of genetically modified T-cells to help her overcome acute leukemia. "We didn't see it as a tough decision. We saw it as the only decision — there was no other option," Layla's mother said in an interview featured on NBC Nightly News in 2015.
The treatment appears to have worked for the little girl. The thing is, she did have other options, according to Annas. Gene editing was just the number her parents bet on.
Richardson's case is reflective of an increasing common question in the gene space: is the benefit of a therapy worth its potential risk. In severe cases in the past, reshaping a man's genome can kill him. Making sure patients, and namely those in the clinical setting, understand such grave consequences has become a notable hurdle for drugmakers and researchers alike.
"The fact that these people are dying, they and their families are demanding something be done," Annas said, adding that, as result of that desperation, decisions such as right-to-try legislation are too frequently debated in terms of emotions rather than scientific evidence. While genetic diseases place an immense toll on patients and their families, it is important to remember that, regarding potential treatments, "the question is when are we ready to try it first in humans," he said.
The "Gene Editing: Promises and Challenges" forum comes at a time when gene-altering remedies are picking up speed to market.
Though there is more work to be done in improving the overall safety of gene editing techniques and the informed consent process, panelists at the forum underscored optimism around innovations in the space, such as the potential in the next few years to use non-human organs for transplants.
"The pig transplant may not work, but what if it does work and that transforms all transplantation?," Howard Kaufman, a member of the National Institutes of Health's recombinant DNA advisory committee, said.
While not always in agreement, Kaufman and Annas conceded that healthcare stakeholders need to be having more conversations about the curative potential of gene therapies, as well as their potentially detrimental side effects.
- Harvard School of Public Health Webcast
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