Evaluating treatment for ill children versus concerning ethical dilemmas

If our variances define our humanity, do we possess the right — or the obligation — to alter them? This inquiry was central to “The Promise and Peril of CRISPR,” a presentation by Neal Baer, Ed.M. ’79, M.D. ’96, co-director of Harvard Medical School’s master’s degree program in Media, Medicine, and Health. During the Science Center lecture co-sponsored by the FAS’ Division of Science and Harvard Library, Baer examined the ethical challenges associated with gene-editing technology alongside Rebecca Weintraub Brendel, director of the Medical School’s Center for Bioethics.

As he introduced the topic — and the book he has compiled under the same title — Baer recounted his “terrible” early experiences treating youngsters afflicted with sickle cell anemia at Children’s Hospital in Los Angeles. “They were in pain. They experienced strokes,” he remembered. “And there was very little that could be done.

“Now,” due to CRISPR, “we have the capability to cure sickle cell,” he stated.

But is it appropriate? This remains the core question for Baer, who is also a screenwriter, dramatizing medical dilemmas in television series like “Designated Survivor” and “Law and Order: SVU.” (“I am Olivia Benson,” he mentioned to the audience, referring to the “Law and Order” character).

With CRISPR technology, researchers can modify both somatic genes (from body cells) and germline genes (from gametes, the reproductive cells that form embryos), literally cutting out and replacing segments of them. In terms of sickle cell, manipulation of somatic cells can, and has, eliminated the genes responsible for the disease in patients. If such alterations were performed in germline cells, the resultant embryo would never develop the ailment.

Yet while remedying this distressing condition may appear wholly beneficial, the inquiries surrounding gene modification generate numerous perplexing ethical dilemmas. “Should we apply CRISPR for diseases or conditions that are compatible with existence, such as Down syndrome?” Baer questioned. “Who is qualified to make these choices?”

The expense associated with gene alteration is another consideration. The sickle cell “cure,” for instance, entails approximately $2.2 million. Pointing out that near 100,000 individuals in the U.S. endure from sickle cell, he queried, “Who will finance it?” The domestic population, he added, constitutes just a portion of the global population suffering, which raises concerns of justice and health equity.

Even if eliminating sickle cell is perceived as a positive, Baer contended, the alterations made feasible by this technology pose troubling concerns. For instance, what if two deaf parents desire their child to be genetically modified to have the ability to hear? “Should parents determine that for their child? Is it appropriate for parents to choose what traits their children should possess?”

In one of the book’s segments, contributor Ethan J. Weiss admitted that had he and his spouse been aware of their daughter’s albinism, “We would have terminated the pregnancy. But now that we have her, we cannot envision a world without her.”

Baer proceeded to quote Carol Padden, dean of social sciences at the University of California, San Diego, who was born deaf. “I don’t have a pathology,” she has articulated. “I embody human variation. I do not require ‘fixing.’”

Another worry is regulation. “Yes, it is unlawful to clone. Yes, it’s illegal to perform germline editing. But who supervises in Russia?” Baer inquired. “Or China?” As if drawing from a television drama storyline, he elaborated on the possibilities of soldiers genetically modified to experience reduced pain, fear, or exhaustion.

Moreover, Baer noted, gene editing might lead to unforeseen ramifications. He referred to a recent breakthrough by the physician and pharmaceutical corporate leader Sekar Kathiresan that enabled him to edit the gene regulating LDL cholesterol. While permanently reducing “bad” cholesterol, which is implicated in heart disease and strokes, seems like a “victory,” Baer cautioned, the science is not straightforward. “This gene has evolved over 3 billion years and plays a role in numerous other” processes, he mentioned, including interactions with insulin and various factors.

“Genes don’t simply vanish,” he asserted. “They evolve and interact and perform multiple functions.”