Gene Editing: Legitimate Therapeutic Tool Or Designer Baby Enabler?

Despite its promise, will gene editing be used as a legitimate therapeutic tool or as a mere way to manufacture “perfect” designer babies?

By: Ringo Bones 

The promise of effectively curing inheritable diseases and the dangers of indiscriminately manufactured designer babies is now a step closer thanks to gene editing – which is an improved version of gene therapy. Techniques to edit human DNA for therapeutic means have been around for some time. One method is gene therapy which was already in regular use for the past 15 years. Gene therapy involves using a disabled virus – known as viral vector – to deliver a synthetic functioning copy of gene into cells. Sadly, this method has a glaring disadvantage because there is no way for scientists to know where the gene will end up amidst the billions of letters of DNA code in the nucleus of cells. 

The latest improved method of “gene therapy” dubbed as gene editing or genome editing offers something far more precise – adding the gene in an exact location. And unlike older gene therapy methods, gene editing can also be used to snip out a faulty section of DNA. 

There are currently several types of gene editing schemes, but the technology really took off three years ago when a cheap and efficient method was developed by Prof. Jennifer Doudna of University of California, Berkeley and Prof. Emmanuelle Charpentier, now at the Helmholtz Center for infectious Research in Germany. Known as Crispr-Cas9, it has been adopted by scientists around the world. 

Crisprs (clustered regularly interspaced short palindromic repeats) are sections of DNA, while Cas9 (crispr-associated protein 9) is an enzyme. They are found in bacteria which they use to disable attacks from viruses. Rather like a GPS sat nav, Crispr scans the genome looking for the right location and then use the Cas9 protein as “molecular scissors” to snip through the DNA. Prof. Doudna and Prof. Charpentier realized that this bacterial cut-and-paste system could be used to edit human genes. Almost immediately Crispr became the gene editing technique of choice because of its speed, accuracy and simplicity. It has caused a quiet revolution in research labs across the world. Experiments which once took months can now be done in weeks and the potential is vast. 

Last month, scientists said they had used it to create a mosquito that can resist malaria. Plant breeders are using Crispr to create disease-resistant strain of crops. Back in April 2015, researchers in Mainland China edited human embryos to try to correct a faulty gene that caused an inherited blood disorder. The embryos used were never destined to be implanted, but the research rang alarm bells about the implications of gene editing – i.e. the “specter” of perfect blond blue-eyed designer babies and editing out mental disorders like manic depression, which unfortunately, is a prerequisite mental disorder for brilliant poets, authors, musicians and playwrights. 

Artificial Intelligence Programs: The New Cancer Treatment Paradigm?

Even though it can’t yet pass the Turing Test, does Berg Health’s artificial intelligent computer program represent a new paradigm in cancer treatment research and development? 

By: Ringo Bones 

Even though the working principles of such computer programs were first mentioned in Star Trek: The Next Generation and other science fiction programs back in the late 1980s, it is only relatively recently that an artificial intelligent computer program had actually reduced the time and costs in the research and development of new anti-cancer drugs - which is of upmost importance if the new anti-cancer drug proves to be safer than current chemotherapy and radiotherapy treatment schemes. Even though it can’t yet pass the Turing Test, it does show promise of reducing the excessive costs and lengthy development times in introducing new and more effective anti-cancer treatment drugs to the market. 

Berg Health – a pharmaceutical start up founded in back in 2008 with Silicon Valley venture capital backing, said that its proprietary artificial intelligent computer program has managed to slash both time and development costs of putting a new more effective anti-cancer drug into the market. It has already managed to develop a new anti-cancer drug that’s expected to go on sale within three years – marking seven years in development compared to the general 14 years using previous methods. 

Recent cancer research shows that healthy cells feed on glucose in the body and then die off in a process known as cell death when their usefulness draws to a close. But in some circumstances the mitochondria – the part of the cell that provides its energy – malfunctions and metabolizes lactic acid instead of glucose, turning off the built in cell death function at the same time. The cell can then become cancerous and a tumor grows. Berg Health’s new drug – BPM31510 – will reactivate the mitochondria, restarting the metabolizing of glucose as normal and reinstituting call death so the body can harmlessly pass the problem cell out of the body. 

Berg Health’s research and development team used a specialized form of artificial intelligence computer program to compare samples taken from patients with the most aggressive strains of cancer, including pancreatic, bladder and brain, with those from non-cancerous individuals. The technology highlighted disparities between the corresponding biological profiles, selecting those it predicted would respond best to the drug being tested. 

“We’re looking at 14-trillion data points in a single tissue sample. We can’t humanly process that”, says Niven Narain, a clinical oncologist and Berg Health co founder. “Because we’re tackling this data-driven approach, we need a supercomputer capability. We use them for mathematics in a big data analytic platform, so it can collate that data into various categories: healthy population for women, for men, disease candidates, etc, and it’s able to take these slices in time and integrate them so that we’re able to see where it’s gone wrong and develop drugs based on that information,” Mr. Narain said. 

Berg Health expects to begin phase two trials of the drug in January 2016, meaning it has already been proven to be effective on animal or cell culture tests and is safe to continue testing in humans. Mr. Narain said it usually takes 2.6-billion US dollars and 12 to 14 years to get a new drug to the market, and that the trial metric within four and a half years worth of development indicated the time it takes to create a new drug can be cut by at least 50-percent. This will also translate into less expenditure, he claimed. “I don’t believe we’re going to spend 1.3-billion US dollars to produce our first drug, so the cost is cut by at least 50-percent too” he added. “There’s a bit of trial and error in the old model so a lot of these costs are due to the failure of really expensive clinical trials. We’re able to be more predictive and effective…and that’s going to cut hundreds of millions of dollars off the cost.”

Should A Breast Cancer Gene Be Patented?

Even though a US firm Myriad Genetics currently holds a patent of one, is it even ethical for corporate firms to hold patents on a breast cancer gene?

By: Ringo Bones 

Recently, a Brisbane grandmother named Yvonne D’Arcy celebrated a High Court victory over the BRCA-1 breast cancer gene patent. Australia’s High Court says “human genes are not patentable invention” as Australia’s High Court overthrows previous ruling. Given the recent court decision, would this spell relief to working class cancer sufferers? 

During the past few years, campaigners are busy raising the issue of gene patenting because if corporations got what they wish for, cancer testing could become prohibitively expensive for working class folks due to exorbitant royalty fees. And every high court the world over not beholden by big multinational medical corporations still subscribe to the view that “corporations can patent something that naturally occurs in the human body”.  

The issue of gene patenting and its implications to social justice, and even medical ethics, had been around since 1992. Social justice campaigners had been concerned when corporate buccaneers became busy prospecting for “patentable biochemical agents” in the Amazon Rain Forest during the early 1990s. If big multinational medical corporations’ got their way, corporations could legally ask for Amazonian tribes to pay them money – as “royalty fees” - every time these tribes use their traditional healing herbs that they’ve been using for thousands of years. 

Daraprim 5,000 Percent Price Rise: A Runaway American Big Pharma Greed?

Does the 5,000 percent price increase of a generic drug a symptom of American Big Pharma greed?

By: Ringo Bones 

Along with Big Coal and Big Oil Capitol Hill lobbyists, Big Pharma is now one of the new symbols of corporate greed that causes unnecessary misery of working class Americans and even people of the rest of the world. And when the drug company Turing Pharmaceuticals recently acquired the license to manufacture a 62-year old lifesaving drug that has since become a generic drug called Daraprim and overnight raised its price by 5,000 percent from 13.50 US dollars per tablet to about 750 US dollars per tablet, millions of people prescribed by the drug are soon up in arms. Even though Turing Pharmaceuticals CEO Martin Shkreli has since roll-backed the 5,000 percent price increase, many now wonder if Big Pharma exploitation of working class patients are causing yet another undue suffering. But is Turing Pharmaceuticals 5,000 percent price rise of a lifesaving generic drug even justified? 

Despite Turing Pharmaceuticals CEO Martin Shkrreli saying that his decision for the 5,000 percent price increase of Daraprim is motivated by altruism, anyone with a rudimentary working knowledge of economics compare the move as akin to a luxury sport-scar manufacturer purchasing the rights to manufacture a South Korean family sedan that used to sell for 6,000 US dollars each and then increasing its price to 70,000 US dollars overnight. All of which only highlights the problem of how corporate greed in America and their super-strong lobbying powers at Capitol Hill is causing undue suffering of Americans and everyone else on the planet. 

Daraprim entered the pharmaceutical market about 62 years ago as a treatment of the parasitic infection toxoplasmosis and has since been priced as a generic drug. Given their compromised immune system, the main users of Daraprim are HIV / AIDS patients who are easily infected by toxoplasmosis and related infections in comparison to people with normal immune systems. 

2015: 60th Anniversary of the Polio Vaccine

Even though measures of eradicating the disease in remote regions of the world still manage to gain headline news status, did you know that the polio vaccine in already 60 years old this 2015? 

By: Ringo Bones 

Back in April 12, 1955, the Salk Polio Vaccine was declared to be safe for public use after years of Dr. Jonas Salk’s dedicated lab-work to eradicate one of mankind’s greatest public health scourges. The still evolving role medical researchers – from cloistered academic eccentric to popular hero – is in many ways reflected in the history of poliomyelitis research which reached a dramatic climax with the acclaim lavished on Dr. Jonas Salk for the introduction of an effective polio vaccine in 1955. But before Salk, researchers in half a dozen countries had paved the way for polio prevention. By the first decade of the 20^th Century, it was known that polio was infectious, was transmitted by personal contact and did not always result in paralysis.

However, the cause of polio had been thought to be bacteria. Then in 1909 – Dr. Sam Flexner, an American pathologist, finally isolated the agent responsible: a virus so small it could pass through the finest filters then in use at the time. But the virus had not yet been found in the bloodstream of its victims. It was assumed that the polio virus grew only in nerve cells and at the time a prototype vaccine made from infected nerve tissue produced side effects sometimes as crippling as the disease itself. Given the extremely small size of the polio virus, even light rays were deemed “too coarse” to see it and thus necessitates the use of electron microscopy which a working one wasn’t invented until 1935. And it wasn’t until 1953 that for the first time an electron microscope allowed human medical lab workers to “see” the polio virus after magnifying it 77,000 times with a stream of electrons – which at the time the most powerful electron microscopes still had trouble seeing objects tinier than 2 angstroms in diameter. 

Forty years after Dr. Flexner’s findings, a team of American scientists was able, for the first time, to grow polio viruses in non-nerve tissue after extensive lab work back in 1949. Other researchers had shown that there were three distinct types of polio viruses which produced identical symptoms and aftereffects. These viruses proliferate in the lower intestinal tract and the throat, causing symptoms so mild that they are often overlooked. Occasionally, however, they enter the bloodstream and eventually attach themselves to nerve cells, resulting in the dreaded paralytic polio. These discoveries provided the theoretical basis for the vaccines for they proved that the disease’s path allows for the production of antibodies that intercept the viruses before they reach the vital nervous system, where they do their damage. 

The first type of polio vaccine which was declared safe for widespread public use was developed by Dr. Jonas Salk and his lab associates at the University of Pittsburg using polio viruses killed by formaldehyde which sets up a protective antibody barrier against polio in the bloodstream. A few years later, Dr. Albert Sabin of Cincinnati developed the first live polio vaccine to be put into regular use and it was licensed for use in the United States back in 1962. Dr. Sabin’s polio vaccine variant generates immunity in both the bloodstream and intestinal track. And widespread global polio vaccination programs soon followed suit where as far back as 1958 a live polio vaccine variant developed by Dr. Hilary Koprowski of The Wistar Institute of Philadelphia which was applied as a mouth spray was used to vaccinate more than 250,000 Congolese citizens. 

It was only in the post-9/11 world that mass polio vaccination efforts became dangerous to the lives volunteer doctors working for charitable organizations after various Islamist Terror organizations around the world started to spread the rumor that the free vaccines used by charitable organizations cause sterility and mental retardation – thus hampering the global eradication of paralytic polio as one of the UN’s Millennium Development Goals. Even as recently as 2003, influential Islamist leaning politicians managed to ban free mass polio vaccinations just by citing the conspiracy theorists’ claims as evidence. It wasn’t only last year that mass free polio vaccinations resumed in the most deprived regions of Nigeria when the Emir of Kano recently took an oral polio vaccine in front of his subjects to show reassurance that polio vaccines are perfectly safe despite of the flimsy claims of conspiracy theorists and Nigeria’s Islamist leaning politicians. Despite its proven safety record for 60 years – conspiracy theorists and even right-wing militant Evangelical Christians in the United States are refusing to have their children vaccinated against polio.