Out of prison, the ‘father of gene therapy’ faces a harsh reality: a tarnished legacy and an ankle monitor

NORCO, Calif. — Dr. W. French Anderson is settling into a leather chair in the family room of his ranch-style stucco home in southern California this June evening, marked-up issues of scientific journals tumbling off piles on the floor beside him. Slowly, he lifts his right leg onto the matching ottoman. He has an hour to kill. That’s how long he must charge his ankle monitor, which tells his parole officer where he is at all times.

This is what it has come to for a world-renowned scientist who was convicted of sexually molesting a colleague’s young daughter. Anderson has been hailed as the father of gene therapy and was honored at George H.W. Bush’s White House. In 1991, the New York Times ran a laudatory story headlined “Dr. Anderson’s Gene Machine.” He started the first gene therapy company and sold it to a major drug maker in 1995 for $325 million, was a Time “hero of medicine” in 1997 and scientific consultant to the 1997 film “Gattaca,” and was inducted into the Oklahoma Hall of Fame in 1998 (with singer Reba McEntire).

But in July 2006, Anderson was convicted of three counts of lewd acts on a child and one count of continuous sexual abuse, including fondling her genitals. The sexual assaults started in 1997 when the girl was 10 and Anderson was 60, prosecutors said, and lasted until 2001 — abuse that his victim testified in court caused her “pain that led me to cut my own body and contemplate suicide.” Her mother ran Anderson’s lab, and he had mentored the child academically and in karate.

Before sentencing Anderson to 14 years in prison, Judge Michael Pastor said he had caused the girl “incalculable” emotional damage: “Because of intellectual arrogance, he persisted and he got away with as much as he could.”

This May, Anderson, now 81, was released on parole. Two weeks later, STAT spoke to him. We were interested in his views — as someone who’d once been near the pinnacle of medical science — of the research advances during his years behind bars, especially in gene therapy and genetics. We wanted to know what became of his own science when he was incarcerated, and whether he planned to return to the lab. But we made clear that we did not plan to re-investigate the criminal case, or provide a platform for his detailed arguments that he was wrongly convicted.

During his 2006 trial, prosecutors played a tape in which Anderson told his victim that his behavior — unspecified in the conversation — was “indefensible” and “just evil.” But over three days of interviews, he offered innocuous explanations for his words, arguing that the tape was doctored and that he was imprisoned for crimes he did not commit.

Anderson was wistful about how science had marched on without him and described scenes one doesn’t associate with prison life: an inmate trying to keep up with genetics, eagerly opening envelopes from his wife stuffed with issues of Science, Human Gene Therapy, and Genetic Engineering News. All along, he said, he was certain he would be released on appeal, and so spent the early years of his incarceration planning experiments he would resume in his former lab at the University of Southern California or with the biotech he had co-founded.

Once the last of his appeals failed in 2012, however, Anderson recognized reality: Job opportunities for an aging ex-con who was behind on the science and hadn’t so much as pipetted in a decade would not exactly flood his inbox.

“I got out of prison and looked at all of the [gene therapy] literature and felt sort of like Rip Van Winkle waking up,” he said. It would be nearly impossible to catch up.

The interviews took place in the three-bedroom house that his wife of 57 years, Dr. Kathryn Anderson, bought to be near the second of four state prisons where he served time. With a model-home feel but far from lavishly furnished, it sits in one of the dozens of planned communities, with names like Riven Glen and Meadowside, that sprawl across Riverside County, interspersed like upscale oases among dusty horse ranches and undeveloped lots. Many of their belongings remain in storage because she moved several times during her husband’s years in prison, and there are no family photographs or other mementos on display.

Anderson was alternately dismissive of science that arrived during his time in prison, including the revolutionary CRISPR genome-editing technology, and animated about the past, which he recalled in near-cinematic detail, from the shyness and arrogance that crippled his boyhood social life — he regularly told other kids in elementary school that he was the smartest one in class — to his clashes with molecular biologists who tried to stop the gene therapy experiment he did at the National Institutes of Health in 1990.

He recounted as if it happened yesterday a 1951 high school debate that vaulted him and his partner into the Oklahoma state championships, races he ran as a Harvard undergraduate, and James Watson (co-discoverer of the double helix, then a Harvard professor) calling Anderson an idiot for choosing medical school rather than graduate school in biochemistry. He recalled the drama and backstabbing from competitors before the gene therapy experiment that vaulted him onto newspaper front pages and secured his scientific legacy — but also turned some colleagues into such bitter enemies that he claims they blackballed him from election to the National Academies.

For someone who maintains his innocence, Anderson showed remarkably little bitterness. In contrast, Kathryn Anderson, 79, who sat through the interviews and provided the occasional name or date or other fact that her husband forgot, seemed more outraged by his incarceration. She went through what she regards as exculpatory evidence with the rigor of the pediatric surgeon that she is — she was surgeon-in-chief at Children’s Hospital in Los Angeles and the first woman president of the American Pediatric Surgical Association — and lambasted a system “where the promotion and livelihood of prosecutors depends on the number of convictions” and there is “not a word about justice.”

Her husband remains proud of his scientific and policy accomplishments, and generous in sharing credit with colleagues and lab underlings, but is seldom given to modesty except when asked directly about his achievements.

“I believe that God gave me certain abilities and it’s my responsibility to use them,” Anderson said, adding that he doesn’t deserve “any credit for it.”

He beamed as he described acing the written test and eye exam for his driver’s license after getting out of prison. Seeming delighted to show he had not lost his athletic ability, he let a videographer shoot his 2-mile morning run, a decades-long passion that he said boosts his mood and clears his head; in prison, when his unit was not on lockdown, he said he got in quarter- and half-mile sprints as well as 2-to-5-mile runs every day.

Even when Anderson summed up his life by saying, “I used to be famous and now I’m infamous,” it seemed matter-of-fact more than rueful. He feels like Muhammad Ali, he said, an icon at the top of his game whose life and career were derailed by what he calls a flawed, unjust system.

He forgot a name or two (“Who was the guy who won the Nobel with [David] Baltimore?”), but otherwise showed little sign that prison had taken a mental toll, portraying himself as largely unaffected, emotionally, by his years behind bars.

“I was never in prison in my head,” Anderson said. “I was in prison in my body. But never in my brain. I never dreamed of prison or anybody in prison. Not one second in 12 years. It didn’t exist for me.”

That was largely, he said, because his wife has stood steadfastly by him since his 2004 arrest. Over the years, she has diligently added to a now 731-page binder filled with what they call evidence of a massive, financially motivated conspiracy to bring him down. Spokesman Paul Eakins of the Los Angeles County District Attorney’s Office, which prosecuted Anderson, said it was “declining to comment further on this case.”

Other inmates warned Anderson that being released and returning to Kathy — whom he spoke to by phone twice a day (wardens put him in charge of the inmates’ phone sign-up sheet) — would be a difficult adjustment. “But we just picked up where we left off,” Anderson said. “It was like I’d been gone for the weekend. … I’m sort of like a dog; I can survive anywhere. Wherever I am, that’s fine.”

If it was bravado, it was Oscar-worthy. He displayed what scientist-entrepreneur Fred Ledley, an acquaintance from Anderson’s gene-therapy days, recalled as a sunny, gregarious personality: “People liked French. I liked French,” said Ledley, a professor at Bentley College in Massachusetts. But “there was also a professionalism to French that was practiced. One of his pieces of advice to people was to get media training. He was skilled at being in the public eye. I doubt there were a lot of unguarded moments.”

‘I was treated as a godfather’

Anderson entered prison reeling from his conviction, but also defiant. He felt certain he would be freed on appeal and quickly return to his USC lab, where in the decade before his conviction he had published 99 scientific papers. It wouldn’t work out that way.

Instead, in his more rudimentary reality, Anderson realized he needed to focus on surviving. In his telling, even as an elderly inmate, he had the speed and physical prowess to counter anyone who threatened him. It was like his experience decades before when, told he was the most unpopular kid in elementary school, he brought all of his budding analytical skills to bear on the puzzle of the preteen social whirl; now, in a confined space where child molesters are viewed with contempt and loathing, he devised another survival plan, starting by establishing himself in the prison pecking order.

At his first prison, in the northern California town of Jamestown, one clique wanted to oust the inmate elected to an advisory council, Anderson recalled. “Big Joe” began going cell to cell asking who dared vote against him. Anderson said he stabbed his finger into Joe’s chest and berated him for, essentially, voter intimidation.

“He says, ‘I’m going to tear your head off, I’m going to do this, I’m going to do that,’” recalled an animated Anderson. “And I’m pounding him on the chest and saying, ‘You can’t do this.’ And all of a sudden he says, ‘You’re right, I’m sorry.’ We shake hands.”

After that, he claimed, inmates gave him their places in food lines and held doors for him. “I was treated as a godfather for the rest of the time, so I was never in danger after that,” he said.

Anderson’s accounts of prison life could not be independently verified. Luis Patino, a spokesman for the California Department of Corrections and Rehabilitation, said information on inmates’ experiences cannot be released.

Later, after he was transferred to the California Rehabilitation Center in Norco, a young inmate threw his fist toward Anderson’s face, he recalled proudly: “Not enough to actually hit me but, you know, clearly threatening.” Anderson defended himself using the karate and other martial arts skills he learned after the 1968 race riots in Washington, D.C. “I just blocked, saluted, nodded, and walked right on,” he said. “They never bothered me again. … When I saw him later, he waved.”

Anderson spent most evenings in the prisons’ law libraries, reading cases that he hoped would support his legal appeals, but with diminishing hopes: “As year five, six, seven became the eighth became the ninth became the tenth,” he said, “by year 11 I finally came to realize, I’m still going to be here” until he became eligible for parole.

The dream died hard. In a 2012 essay from prison that his wife posted on Anderson’s now-defunct website, he wrote that his “intention, once I am exonerated, is to return to developing improved gene therapy products for the treatment of cancer.” What he missed most, after his wife, he said, was “the ability to be creative. I kept trying to write out experiments I was going to do when I got out. But I never got out.”

There were smaller rewards. At Jamestown, he tutored inmates for up to eight hours a day. “Some were so dumb, all we could do is show them alphabet cards and try to get them to recognize what the letter C looked like,” Anderson said. “Many of the people in the class couldn’t care less. But many did care.” When one young inmate passed his high school equivalence test, Anderson said, “He leaped in the air and said, this is the first thing I ever succeeded at in my life.”

History ‘as complex as French is’

Even before his arrest, Anderson evoked starkly different reactions from his peers, especially after he started laying the foundation for what he was determined would be the first approved gene therapy experiment.

Although Anderson became known as the father of gene therapy, with all the public recognition and accolades that brought, it was not because of any scientific breakthroughs. Gene therapy then, and now, involves slipping a healthy gene into millions of innocuous viruses, which carry the gene into the cells they infect. The girls in the historic experiment had ADA deficiency, which causes severe combined immunodeficiency, or “bubble boy disease.” Anderson neither developed the crucial viral vector nor isolated the healthy ADA gene. Nor did he ever claim to.

“The history of that experiment is as complex as French is,” said Dr. R. Michael Blaese, a retired NIH immunologist and expert on ADA deficiency who found the girls and collaborated with Anderson. “He got into conflicts with a lot of people. French would hear about things, and if they were successful, he’d incorporate them into his own inventions. … French’s contribution was that he understood the politics and the regulatory environment. He knew the players who had to be convinced. And he was the one who had the dream.”

Indeed, as early as his undergraduate days at Harvard, Anderson told his academic adviser that he intended to cure inherited disease by giving people healthy replacement genes.

Anderson and his colleagues spent much of the 1980s trying to get viral vectors to carry repair genes into stem cells that would infiltrate bone marrow, first in lab dishes and then in monkeys. “They were never able to get the human ADA gene expressed in bone marrow,” Blaese said. Anderson was nevertheless determined to make his dream a reality, and so in 1987 lugged a phonebook-sized proposal for a gene therapy experiment to the NIH panel that had been recently formed to vet such research.

Without convincing data from animal research, the panel basically said, “It’s a very exciting idea. No, thank you,” Blaese recalled. The molecular biologist members were almost united in opposition, regarding Anderson with disdain bordering on contempt, as a “dabbler” who threatened scientific integrity by pushing for a human experiment before anyone knew what would happen to introduced genes.

Anderson and Blaese’s next experiments, slipping healthy genes into white blood cells, went better. But the NIH panel still balked and asked for more experimental data. Even when it finally OK’d the experiment in 1988 by majority vote, the NIH director said no; a step of this magnitude needed near-unanimity, recalled Nelson Wivel, the former NIH official who oversaw the gene therapy panel.

Through all the pushback and demands, “French was unflappable,” Wivel said. “He always provided the additional information. Without his total and complete dedication, that first protocol wouldn’t have been approved. He was relentless. He could respond to critiques without losing his temper.” The gene therapy trial was approved.

All these years later, Anderson clearly remembers the battles he fought. “Gene therapy isn’t just a scientific problem,” he said. “It’s also an ethics problem. It’s a regulatory problem. What I accomplished, which I am proud of, is that I worked on all of those problems simultaneously. I’m the one” who got it done.

In contrast, what Anderson calls “that clique” thought he was cutting corners by not proving the safety and efficacy of gene therapy on non-human primates before trying it in children.

“This group of Cold Spring Harbor Lab and Harvard molecular biologists basically thought it was just wrong, dangerous, irresponsible,” Anderson recalled, warming to his topic. “And I, as the leader of the gene therapists, was reckless, irresponsible, etc. I wanted to do something they thought was impossible. I did it anyway and it worked. The last one was what they couldn’t forgive.”

‘It’s just evil’

After Anderson’s last appeal was denied in 2012, he vowed to work for criminal justice reform. But over a takeout dinner of KFC and homemade green salad with mandarin oranges, he conceded that that’s unlikely.

“My plan had always been to be the face and the voice for the faceless and voiceless victims of the criminal justice system,” he said. “I would have a bully pulpit: I was famous enough, and now infamous enough. … To finally prove my innocence would be a major story. But any [reversal of his conviction] is probably going to take a couple of years, so we will see.”

For now, at least, Anderson’s world has shrunk to his wife, his running and karate, his reading, and all-consuming quest to prove his innocence. A few days before this story was published, Anderson emailed a three-page document he had written, called “Highlights of the Forensic Evidence That Established the Malevolent Alterations Made in the Sting Meeting Recording.”

As he has for years, he insists that an audiotape played at his trial (his victim wore a wire when she confronted Anderson in July 2004 near the South Pasadena public library) was doctored by a detective to make it appear that Anderson admitted abusing her. He doesn’t explain why authorities would want to frame him, mentioning only past corruption convictions of officials in the Los Angeles County Sheriff’s Department.

The tape was full of damning statements. He called his (unspecified) interactions with the girl “indefensible.” At another point, he said, “I can’t explain it. It’s just, it’s just evil.”

The Andersons argue that those statements and others refer to pushing the girl too hard academically and in the sports, including karate, that he coached her in. Choosing not to have children themselves, they had mentored nine boys and girls over the years, including two who testified for him at his trial. STAT was unable to contact the victim; her mother, whom Anderson hired to run his USC lab, did not reply to interview requests.

It was not only the audiotape but also emails that helped convict Anderson. In response to the girl’s emailed request for an apology, for instance, he wrote that he “can understand what would drive a person to suicide. For me, a powerful 9-mm bullet through the head would be the way to go” and “just in case, I have bought the ammunition.” In another email, he wrote that he “came to the sad conclusion that there must be a very bad part of me that, now that I have recognized it, has to be permanently suppressed.”

A kitchen lab

Surprisingly, perhaps, the success of the 1990 gene therapy experiment is still debated, foreshadowing the polarized views of the last science that Anderson did before his arrest.

The two girls who received gene therapy remained on their ADA drug, leaving unanswered how well the repair gene worked. Even the treatment’s safety did not unlock the floodgates. The death of a patient in a different gene therapy study run by scientists at the University of Pennsylvania, and difficulties regulating inserted genes, made the field a backwater for years. The work that vaulted Anderson to fame and fortune was essentially a one-off.

And although in 2017 the Food and Drug Administration finally approved the first gene therapy, which was modeled on Anderson’s 1990 procedure, it seems like a throwback, likely to be eclipsed by the more precise techniques of genome-editing such as TALENs and CRISPR.

Anderson dismissed the seminal CRISPR papers that appeared in 2012 and 2013 as “not that surprising.” After all, he said, when scientists discovered DNA-snipping enzymes called restriction endonucleases in bacteria in the 1970s, some suspected bacteria had something like CRISPR (an anti-viral defense system), “but of course they didn’t [call it that]. We all knew genetic editing technology was there [in bacteria]. It might take 50 years but somebody was going to find it.”

Even as he described the discovery of CRISPR as the inevitable outcome of decades-old research, however, Anderson recognized its potential to make the sort of gene therapy he aspired to — put healthy genes into viruses, inject the viruses into a patient — as passé as medical leeches. With CRISPR, he said, “it might be that gene therapy becomes obsolete.” That, he said, would be fine with him.

Before his sentencing, some 200 prominent scientists and other colleagues calling themselves “Friends of French Anderson,” wrote letters to the court vouching for his integrity and character. But now, most scientists contacted by STAT declined to address his conviction, explaining, as one put it, that “there’s no upside to being in a story about a convicted child molester.”

Still, as Anderson’s legacy fades into history, some scientists still regard him as a pioneer. On the day of Anderson’s release from prison, Dr. Jean Bennett of the University of Pennsylvania asked the audience at a talk she delivered at the annual meeting of the American Society of Gene and Cell Therapy to “please welcome the father of gene therapy, Dr. French Anderson … back in the world of freedom!”

She described how Anderson “inspired and led the way for me and many other people to go forward to test gene therapy treatments for devastating genetic diseases. … I hope that people will welcome him back and help him catch up.” Her request “received a standing ovation,” she said in an interview, a tribute “to the compassion and persistence of all of the investigators [in gene therapy], beginning with French.”

Anderson’s later work, much less known, awaits the verdict of history. At USC, he was developing a drug based on the protein IL-12 with funding from a biotech he co-founded, Farmal Biomedicines. (The name comes from the initials of French Anderson and Robert Monks, a Harvard friend and the main investor, together “at last.”) In 2003, he and his colleagues discovered that IL-12 protected mice from what would otherwise be a lethal dose of radiation, including if given after exposure.

That could save victims of a nuclear accident, Anderson said, but it might also let cancer patients safely receive double or triple the standard radiation dose, killing more cancer cells without causing radiation damage. A biotech he co-founded after Farmal imploded, Neumedicines, continues to develop IL-12 under a license from USC, said CEO Lena Basile.

Anderson was also pursuing gene therapy for cancer, using viruses to ferry “sleeper” suicide genes into tumor cells: The idea was that a simple drug would activate the genes, killing the cancer cells. The key step was creating mutant forms of the Moloney virus, which infects mice but can be tweaked to infect human cells; he was seeking one adept at penetrating cancer cells but sparing normal ones.

“I had about 80 mutants all characterized, sitting in the freezer ready to go,” Anderson said. “But that was when I got arrested. So it just collapsed. I hadn’t published the concept [of how to do make the Moloney virus carry suicide genes into cancer cells]. It’s still in my head, but that doesn’t do much good: I’m 81 years old and don’t have a lab. But we were close.”

Researchers who followed Anderson’s work expressed doubt about that claim, with one saying “he was out of his depth,” and another saying, “It didn’t seem like there was much science to what they were doing.” Like most scientists contacted by STAT, they did not want their names used.

As the years passed and Anderson’s hopes of returning to science receded further and further, he said, “I kept narrowing down the experiments I was going to do. Finally I’m down to having my wife’s kitchen. That’s all I have for a lab.”

He brims with excitement, though, about the experiment he dreams of doing there. “It follows up on an observation I made in 1966,” when he worked in the NIH lab of Marshall Nirenberg, who shared the 1968 Nobel prize in medicine for deciphering the genetic code. When Anderson warmed up a solution of the paired DNA bases G and C in a test tube, he recalled, it became solid, indicating the DNA building blocks had formed bonds. Cooled, it became liquid.

That long-ago observation has intrigued him ever since. He wondered whether heat could allow the two bases to form a double helix like DNA. If so, “that is the major step in the origin of life,” Anderson said. “I want to test that. It’s a simple experiment to do, you just buy the molecules and heat them up and see what happens.” 

It’s a quixotic notion, that he could discover something so fundamental amid the pots and pans. But that’s about all the science he can hope to do, with his ankle monitor due for charging this evening.