First Data from Anti-Aging Gene Therapy

A biotech company reports that an experimental treatment elongated its CEO’s telomeres. 

By | April 25, 2016

FLICKR, HEY PAUL STUDIOSLast year, Elizabeth Parrish, the CEO of Seattle-based biotech firm BioViva, hopped a plane to Colombia, where she received multiple injections of two experimental gene therapies her company had developed. One is intended to lengthen the caps of her chromosomes (called telomeres) while the other aims to increase muscle mass. The idea is that together these treatments would “compress mortality,” Parrish told The Scientist, by staving off the diseases of aging—enabling people to live healthier, longer.

On its website last week (April 22), BioViva reported the first results of Parrish’s treatment: the telomeres of her leukocytes grew longer, from 6.71 kb in September 2015 to 7.33 kb in March 2016. The question now is: What does that mean?

The company announced Parrish’s response as success against human aging, having “reversed 20 years of normal telomere shortening.”

Over the phone, Parrish was more measured in discussing the implications of the finding, which has not yet undergone peer review. “The best-case scenario would be that we added 20 years of health onto the leukocytes, and the immune system might be more productive and catch more of the bad guys,” she said. “But we have to wait and find out. The proof will be in the data.”

Much more data are needed before claiming success against aging, said Dana Glei, a senior research investigator at Georgetown University. “We haven’t established a causal link between telomere length and health,” she told The Scientist. “If it’s like gray hair, dyeing your hair won’t make you live longer.”

An n of one won’t give us the answer, but Parrish’s personal trial is the start of what BioViva hopes to accomplish: the first clinical studies using a gene therapy to stall aging and increase health span.

The company’s approach is backed by preclinical evidence—in particular, that from María Blasco’s group at the Spanish National Cancer Research Centre (CNIO) in Madrid. In 2012, Blasco’s team reported the results of a telomerase gene therapy in mice. The enzyme telomerase, encoded by the TERT gene, lengthens telomeres.

“We demonstrated that AAV9-Tert gene therapy was sufficient to delay age-related pathologies and extend both median and maximum longevity in mice,” Blasco, who is not involved with BioViva, told The Scientist in an email. “Many pathologies were delayed, including cancer.”

Blasco’s team has since demonstrated that telomerase gene therapy can abate certain age-related diseases in mice as well.

Some human diseases are the product of shorter-than-usual telomeres, noted Abraham Aviv, who studies the chromosomal caps at Rutgers New Jersey Medical School. “However, the idea that in the general population relatively short telomeres are bad and relatively long telomeres are good is nonsense,” he wrote in an email.

Telomere length is associated—in opposing directions—with cardiovascular disease and cancer risk. “I refer to this phenomenon as the cancer-cardiovascular disease trade-off, which largely defines longevity of contemporary humans,” Aviv added.

And telomere length is not a good predictor of mortality. Earlier this month, Glei and her colleagues looked at how well leukocyte telomere length correlated with dying within five years. After adjusting for age and sex, they found that more than a dozen other measurements—from self-reported health status to C-reactive protein levels—were better at predicting five-year mortality.

Aviv pointed to another potential weakness of the BioViva data: measurement error. The 9 percent difference between Parrish’s before and after telomere lengths is “within the measurement error of most laboratories,” he said. “Thus, I am not impressed by the difference between these two values, based on one case.”

Houston-based SpectraCell Laboratories conducted the telomere length assay for BioViva. Jonathan Stein, the director of science and quality at SpectraCell, told The Scientist that most telomere-length assays have a variance of 8 percent, and his firm’s test is in line with that number.

The other gene therapy Parrish received—the gene encoding the follistatin protein—is supported by human data, at least in the context of people with muscle disorders. (There are not yet data demonstrating the effects of follistatin gene therapy on aging-related muscle loss.) Follistatin inhibits myostatin, which puts the breaks on muscle growth and therefore makes it an attractive therapy for muscular dystrophies. Early clinical trials on six people with Becker muscular dystrophy, for instance, showed that four of them could walk longer distances after the follistatin gene therapy. Parrish said she expects MRI data on her muscles’ response to the treatment in about a month.

“Translating these results to human diseases (telomere syndromes or certain age-related diseases without effective treatments) may be of interest in the context of clinical trials approved by the corresponding regulatory agencies,” Blasco said.

Working with regulatory agencies has been a sticking point for BioViva, hence Parrish’s trip to Colombia. Her controversial move—to skirt oversight by the US Food and Drug Administration by receiving the gene therapies outside the country—prompted a member of the company’s advisory board, the University of Washington’s George Martin, to resign, MIT Technology Review reported in October.

Parrish said she is now traveling the globe to find a regulatory partner willing to approve human clinical trials. “When I started looking into this, it seemed like a crazy science,” she said. “But it’s a crazy science whose time has come.”

Parrish’s goal of extending health span, however, is anything but novel. From medicine to meditation, researchers have been looking for ways to keep people healthy as long as possible. And we do have some proven interventions, Glei pointed out.

“My conclusion would be, if you want to live longer you’re much better off smoking less and exercising more than you are trying to lengthen your telomeres,” she said.

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Avatar of: dobermanmacleod

dobermanmacleod

Posts: 1

April 26, 2016

I have been going around and around with other members of the radical longevity community about truth.  I have made the provocative claim that Liz Parrish might be the first immortal human.  Other scientists are outraged by my statement - how dare I make such an unsupported claim!!   On the other hand, the postings to forums on Facebook prefaced with that question got many more hits and likes and shares.

Many of us are having a great deal of difficulty understanding why the regulatory agencies of the US are so risk adverse when willing and informed volunteers who have fatal disease want to undergo experimental procedures to potentially save their lives.  Perhaps we will have to go abroad to circumvent the onerous bureaucracy the US has built up.

When has the Hypocratic Oath to do no harm come to mean take no risks?  Senescence is a disease that kills more people than any other, yet treatments that show promise are kept on the slow track for fear of harming us?  Let's take some chances, try to save some lives, and stop hiding behind the conservative bureaucracy of the status quo.

Avatar of: LAH

LAH

Posts: 5

April 26, 2016

There is no contract that can completely protect scientists from the liability of a clinical trial gone wrong. Many folks on Facebook do not have a viable understanding of scientific principles. but may be all too ready to sue. Even though the AIDS campaigm was sucessful on speeding up the path to viable medications, scientific principles were not abandoned. Just because Ms. Parrish has longer telemeters does not make her immortal. Obviously you have a tenuous hold on reality, dobermanmacleod.

Avatar of: dmarciani

dmarciani

Posts: 38

April 26, 2016

Apparently the assumption is that extending telomers is the solution to stop the normal process of aging, which unavoidable ends in death. Yet, the aging process is not dependent in one or two genes, but it is more complicated; we should not forget that during life we accumulate a series of damages to our DNA, which eventually will impact our bodies. Maybe, it would be wiser if before trying to extend life, we may sure that we can prevent neurodegenerative diseases like Alzheimer’s; otherwise we would end with semi-immortal but brainless people, between other things.

 By the way being an older scientist I have seen about 40 years ago cancer being successfully cured in mice, later I have seen Alzheimer's successfully treated in mice; the downside is that we have been successful in treating mice but unsuccessful in treating man. Hence, I would not scream "eureka" because mice are responding as usual quite well. 

Avatar of: SteveM

SteveM

Posts: 1

April 26, 2016

The U.S. FDA, and other regulatory agencies around the world, are using antiquated laws to police a cutting-edge technology. If companies follow the current law, we will not see gene therapy solutions for deadly afflictions any time soon. The alternative, that BioViva and others will certainly pursue, is to discard the broken web of laws governing what people can and can't do with their own DNA. Even if that means moving to a more proactive climate outside of the U.S.

Here's the bottom line: it's your DNA. If you are dying of a disease with a 100% mortality rate, would you turn down even a small chance at beating the disease via an experimental treatment?  And, even if your particular choice is certain death, isn't it still valid that others may have different opinions about their own medical treatments? I think that anyone who believes that an individual's life-or-death choices should remain in the hands of a government agency is part of the problem. And it's simply a matter of time before many people realize this same conclusion.

Self-expermentation will harm and, likely, even kill people. But many FDA approved drugs do that already. Self-experimentation may also result in break-throughs that have positive consequences for future treatments to many diseases. And really, it's that last point that trumps all other arguments.

Steve

 

Avatar of: symcoxkd

symcoxkd

Posts: 2

April 27, 2016

SteveM, your point might have some validity if we did not live in a society with insurance.  You do not have the right, in my opinion, to spend society's money to purchase your therapies that are considered ineffective.

The next step in the typical argument is that you have the right to the therapy, therefore the government or private insurers must pay for the therapy.  Since this has cost to the rest of us, we have a say in whether to waste our money on a therapy that doesn't work, or hasn't proven itself.

 

Avatar of: JOHN R. GIBBS

JOHN R. GIBBS

Posts: 1

April 30, 2016

I would love to be part of this study. I have had C-3,45 and L45 replaced with lordic cage in Lumbar and titinium plate in C spine. Also titainum plate with bone graft in Left Clavical. Have had Left Kidney, 1/4 liver, Left Elbow rebuilt, spleen removed, almost every bone broken at some point and have an invention that is a body suit that is controlled by wireless hat that  uses the already availible nerves in Quadroplegics and paralized patients that would give them their ability to move. It is lightweight and like a wet suit that surfers wear. My grand mama is Columbian, Papa from Panama Sister from Cuba and my Grandfather worked on the Pamama Canal Zone. I keep up with the scientific developments in the medical feild and have had almost every kind of test that a man can have. I have Osteoporosis at 55 and Osteoperia and a long list of problems that I deal with. I went threw kemo for Hep-C with experimental drug and was first to be healed of it. Dr Ema Ocampo at Tampa General Health park was my doctor. She said that instead of taking interferon injuction and 12 pills a day ( injection of interferon was once a week0 but now just 2 pills a day with plenty of water. I never drank so much water before and I worked construction before I broke my back in 1999. Anyway I truely think this will work. Like if a person who is already paralized would have the danaged nerve endings where the trama accured cut out and a stem cell incerted into the nerves at both ends like a connector in a car for instance them there would be a free passageway for the electrical curent from the nerves or even a small computerised component in my siut will give the extemities movement since it will be coming from their own brain. I have trained my Left brain to work both sides of my body threw thinking real hard. The neurologist said my brain must jump over the dead places to get a live wire. I use to be paralized also. Anyway this is very promising in many ways....

Avatar of: SistuhMoon

SistuhMoon

Posts: 1

April 30, 2016

I believe we will be hearing a lot more about telomeres and telomerase moving forward. Truth is, they are a very big deal.

http://www.agingorganic.com/2016/04/29/the-holy-grail-of-aging-how-long-are-your-telomeres/

Avatar of: hasan1

hasan1

Posts: 1

May 3, 2016

i hope that thjis really does work....sign me up...i am one who wish to live forever if possible....by any means period...life is good death is truly a mistery

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