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How is biotech combating these five killer diseases on the planet?

Copyright © DrugPatentWatch. Originally published at How is biotech combating these five killer diseases on the planet?

Biotechnology is a massive scientific field that uses research tools from biology and chemistry to solve problems and that includes human diseases too. Ever since its existence, it has revolutionized mankind by contributing a lot towards the growing global and public health needs. It provides effective diagnostics, prevention and treatment measures, which includes production of recombinant vaccines and novel drugs for some killer diseases.

Life quality, health and expectancy of life have greatly been enhanced through the services provided by biotech worldwide. Infectious and parasitic diseases like tuberculosis and AIDS have been diagnosed rapidly at a cheaper rate. Molecular diagnostic tools like polymerase chain reaction (PCR), monoclonal antibodies and recombinant antigens have been used for this purpose. Vaccines produced from biotechnology have eliminated polio, small pox and other killer diseases so far. Biotechnology has made brilliant developments in vaccination by producing recombinant vaccines, which have the potential to wipe out non-communicable diseases, such as cancer.

With US biotech revenues touching $72 billion in 2013, some believe that biotech could become the next tech bubble. According to Statistica, over 40% of the world’s share of filed biotechnology patents was made between 2010 to 2012.

This article sheds light on how biotechnology is being used to fight 5 of the top killer diseases in the world. Although it may still take a couple of years to eradicate these diseases using this technology, it’s still a novel advancement in the field of biotechnology.

  1. Pneumocystis

Pneumonia, an infection of the lungs, kills nearly 1 million children under the age of 5 every year worldwide. Even in developing nations like the US, approximately 1 million people seek hospital care each year due to pneumonia. Unfortunately, over 50,000 die from this lethal disease in the country alone annually. And most of the people affected by pneumonia in the US are adults. It’s an illness that can rapidly see deterioration in the health of those unlucky enough to contract it.

In April 2016, researchers at the National Institutes of Health Clinical Center along with foreign organizations sequenced almost the entire genome of human, rat and mouse Pneumocystis. This organism causes lethal pneumonia in immunosuppressed hosts. In fact, Pneumocystis was the first infection that led to the recognition of the HIV/AIDS epidemic. As such,  the disease remains a significant risk not only among the HIV/AIDS population, but in immunosuppressed patients and transplant recipients too.

By analyzing the genomes, researchers have now come to know where the organism actually lives and how it prevents eradication by the host’s immune system. The genomes not only recognized the metabolic pathways that are critical to the growth and survival of the organism, but also identified major pathways that are present in other closely linked fungi, which are not present in Pneumocystis. It was found that the pathways disappeared as Pneumocystis developed solely dependent on its host to survive.

Joseph Kovacs, M.D, senior investigator said that the study is an important step forward in the hunt to know more about Pneumocystis infection and to shrink its impact on immunosuppressed humans.  The elaborate description of these genes that exist or are absent should help attempts to culture the organism, an important breakthrough in Pneumocystis research. Culturing will allow the screening of a huge number of drugs to accelerate identification of innovative treatments for the killer disease.

  1. Ebola

Medical experts around the world are of the opinion that it was inevitable for Ebola to make its way across the Atlantic to the US. The disease was first identified in 1976 but it was on a small scale. The latest outbreak in Africa has claimed over 11,000 deaths, with 30,000 people being infected. It’s a rare but lethal disease and is transmitted from human to human through contact with the patient’s bodily fluids or with items like bedding, that’s normally contaminated with the fluids. The span of incubation lies between 2 to 21 days and patients aren’t infectious before the symptoms develop. However, they can remain contagious for as long as seven weeks after recovery.

Currently, there aren’t any medications for treating Ebola. So treatments mostly include palliative care like treating the symptoms of the disease and rehydration. Still both big and small researchers are working on it.

Tekmira, for instance, has garnered a huge media attention following the use of its experimental vaccine to treat a doctor who had contracted the disease in Africa and was returning to the US. The FDA also granted permission for emergency use of the drugs for patients who had contracted the disease. Thanks in part to the grants of $140 million from the Department of Defense (DoD).

However, Tekmira isn’t the only biotech company that has received money from the DoD. Sarepta Therapeutics, a biopharmaceutical company in Massachusetts, received $291 million in 2010 from the DoD to develop AVI-7537, a drug in development intended to treat Ebola. The drug works by intruding with RNA-signaling involved in protein synthesis. Simply by changing protein synthesis, the medicine can change the molecular structure of developing bodies, eventually halting or slowing the progress of Ebola.

GlaxoSmithKline is also working with the National Institutes of Health on another Ebola vaccine that has shown promising results in pre-clinical trials. The company currently finished their Phase II study where recipients of the GSK vaccine developed an antibody response to Ebola.

  1. Rabies

After you get a bite from a rabid animal, there’s no way to know whether the animal has transmitted the virus to you. And even if the infection is established, there’s no effective treatment for now. Though a tiny number of patients have survived this disease, it’s normally fatal. According to the WHO, over 59,000 people worldwide die from rabies annually. Rabies is caused by a virus that affects the central nervous system, causing inflammation to the brain. Domestic cats, dogs, rabbits and wild animals like raccoons, skunks and bats are able to transfer the virus to humans through scratches and bites.

The rabies virus has been reported from all continents barring Antarctica. It can be cured if patients are given rabies globulin or vaccinated after the bite. Even then, one victim every nine minutes die from rabies, mainly in Africa and Asia. And more than 40% of people bitten are kids under 15 years.

German-based biotechnology company, CureVac, recently published proof-of-concept data from their Phase I trial of a mRNA-based drugs to treat rabies. The drug (CV7201), encoding the rabies virus glycoprotein, was accorded the International Nonproprietary Name (INN) Nadorameran by the WHO as the first drug entity belonging to this class.

For the first time the study demonstrated that the drug was safe with a decent tolerability profile. It also demonstrated that a mRNA-based vaccine candidate can easily induce functional antibodies that can be boosted against an aggressive antigen when injected with the help of a needle-free jet injector.

Although there’s a need for greater research, the results are a vital first step towards realizing the potential of this form of treatment.

  1. Cancer

The field of immuno-oncology is roaring with billions of dollars in investment. The ability to revamp our own immune system to combat cancer has created massive expectations. Following the success of the maiden checkpoint inhibitors on the market, many companies are now focusing their attention to CAR-T therapy-a biotech cancer therapy to hit the market. After the FDA approved Novartis’ CAR-T cell therapy Kymriah(TM) (CTL019) for children and adults, there are over 300 CAR-T clinical trials running currently.

A CAR-T therapy consists of gathering T cells through apheresis, a technique that withdraws blood from the body and then removes one or more blood components (like white blood cells, platelets or plasma). The rest of the blood is then returned into the body. The T cells are sent to a drug manufacturing facility or a laboratory where they are genetically engineered to create chimeric antigen receptors (CARs) on their surface. Following this process, the T cells are referred to as CAR-T cells, where CARs are proteins that permit the T cells to identify an antigen on targeted tumor cells. The CAR-T cells are then infused into the patient.

Clinical trials have shown massive remission rates of up to 94% in acute forms of cancer, which is pretty impressive taking into consideration that most of the trials recruited patients who did not respond to other available forms of cancer treatment. These results have fed the expectations of investors and patients alike, but, researchers caution that it’s still early days for CAR-T cells including questions like whether they can effectively treat solid tumors like colorectal and breast cancers.

  1. Mad Cow Disease

The original outbreak of mad cow disease affected only a tiny number of people with a specific genetic signature. Today, many stringent, control measures have nearly eradicated the danger of infections through contaminated food. But can we shun these infections from reoccurring? Even now, one in every million develops a prion disease like Creutzfeld-Jacob disease (CJD) or a variant of CJD.

Mad cow disease results in a spongy degradation of the spinal cord and brain in cattle and humans who are infected and can develop CJD. The disease came to the public attention in the mid 1990s in the UK killing roughly 80 young people before it was contained. However, slowly the hysteria made its way to the US and 223 people were diagnosed with CJD in the world.

The symptoms of prion diseases develop after years or even decades in some cases. This is why patients who are unaware of the disease can spread it unknowingly while donating blood, for example.

Scientists recently identified prions via examining bacterial genome sequences. They used modeling techniques to predict that a protein in Clostridium botulinum, a bacterium, could take on the prion. The main role of the specific protein is in gene expression, indicating it controls how genetic material is turned on and off. Now that the first example of a bacterial prion has been known, others are almost certain to be found. The findings add a new dimension to how we think about proteins, genes, and possibly the cure for the disease in both humans and animals.


Lethal diseases are still everywhere and this won’t change for some years to come. However, propelled by recent major outbreaks, promising approaches towards cures and prevention have been identified. Still, greater scientific efforts are required to create efficacious therapies and vaccinations that can be used in affected regions.

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Copyright © DrugPatentWatch. Originally published at How is biotech combating these five killer diseases on the planet?
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