Breakthrough Viral Gene Therapy Cures Hemophilia, Possibly Cancer

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Breakthrough Viral Gene Therapy Cures Hemophilia, Possibly Cancer

Photo by: Maria Burmistrova via Shutterstock


The revolutionary, prospective means of treatment in the 1980s was this cutting-edge, newfangled thing called gene therapy, which is just therapeutically administering nucleic acid to the cells as a drug to fight illness. Today, that mode of treatment is in no way prospective. Not only has it come to fruition but it also has complexified with the use of all manner of recombinant viruses as vectors in addition to the naked DNA administration just mentioned. Viruses give their genetic material to a host cell when they get in so they can replicate and thereby deceive host cells into manufacturing more virus. Now, human beings are fighting fire with fire to use viruses to combat disease.

This has become one of the primary avenues of research for dealing with a lot of the diseases that present the greatest hurdles for human health like cancer. It’s because viruses are now seen by the medical science community at large as, among other things, “gene shuttles,” as the expert authors of a new study call them. These expert authors at the University of Zurich just reconfigured an adenovirus for cancer therapy. They made a new protein shield that conceals and protects the virus from eradication, and they engineered adapters on the virus’s surface that enable it to infect tumor cells.

Viruses are too numerous to count, but human adenovirus 5, in particular, has proven to be considerably beneficial in Western medicine. This one just gives you common cold symptoms, but its genome can be entirely supplanted with an artificial genome that only involves advantageous genes. It’s a way for human beings to do within their own bodies the same thing that viruses do except in a way that benefits us. When using this method to treat a virus, the virus can’t continue thriving because all the viral genes have been removed.



While progress is being made on the cancer front, this method has proved so effective as to finally produce an actual cure for hemophilia. A private biotech firm in the US called BioMarin has partnered with Sheba Medical Center based in Ramat Gan, Israel for the next phase of international trials for a cure they’re calling a “magic bullet” for hemophilia A. The epidemiology of hemophilia shows that 1 in every 5,000 males have hemophilia A in particular, and this, of course, refers to the male population worldwide. As such, there are massive swaths of every population that can be positively affected by this groundbreaking research.

“People in the medical community are beginning to speak about curing hemophilia after a one-time treatment with this new drug,” according to Prof. Gili Kenet, Sheba Medical’s director of the National Hemophilia Center and Institute of Thrombosis and Hemostasis. She’s coordinating the research project on Israel’s end, and the attention that the project is receiving now only suggests positive things for the future. New England Journal headlined its coverage of the new breakthrough in progress, “A Cure for Hemophilia within Reach.” Mirroring them, Forbes expounded under the headline, “Excitement Builds around Gene Therapy Cures.”



The excitement comes from how definitive the research is and how conclusive the results have been thus far in previous trials. Says K. John Pasi of the London School of Medicine and Dentistry and Barts Health NHS Trust, “The easiest thing to say is that we were absolutely blown away by [the results].” Pasi led the study to this point for BioMarin in California. The patients were actually able to stop treating their hemophilia with clotting factors and still collectively reduce their average bleeding rates from 16 episodes a year to only one. “Their bleeding rates collapsed to zero or nearly zero and we’ve improved their quality of life beyond recognition.”

One of the pharmaceutical researchers involved, Glenn Pierce, remarks on the profundity of these results because he used to have hemophilia and had to have a liver transplant due to complications with hepatitis C. “It’s a remarkable gift to be able to go from thinking about hemophilia every minute of every day to no longer worrying,” Pierce explains. Hemophilia, mind you, isn’t just feared for the large numbers affected by it but much more for how terrifying it actually is to have. Mere cuts are life-threatening for hemophilia patients.

“If I stand up from this chair and I feel a little pain in my ankle,” Pierce elucidates, “I know it is arthritis from all my bleeding episodes. It’s not bleeding. It’s a remarkable change in outlook, in independence, in freedom, to be able to now function without having to think of my hemophilia. That’s what a cure will mean for individuals who are able to achieve that through gene therapy.”


Photo Credit via Pixabay


This also reinvigorates the hope that some similar breakthroughs with viral gene therapy will come down the pike for cancers as well, which is what that research team in Zurich is endeavoring to achieve. They’re using a new protein shield to both hide and safeguard viruses so that these viruses can carry genes to target tumors. They intend to make novel therapies out of this process to adopt this stealth gene shuttle method to cancer treatments. It’s anticipated that the aforementioned benefits associated with using adenoviruses will serve as a biotech solution to the drug resistance problem. Cancer medicine has, from day one, been thwarted more than anything else by the ability of a cancer to develop resistance to a drug.

“With this gene shuttle,” comments Andreas Pluekthun, a biochemist who worked on their latest study of this biotech method, “we have opened up many avenues to treat aggressive cancers in the future since we can make the body itself produce a whole cocktail of therapeutics directly in the tumor.”



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