These diseases were once considered untreatable

By Naveed Saleh, MD, MS
Published May 27, 2021

Key Takeaways

You can never count out human ingenuity. So was the lesson we learned in December when the first COVID vaccines were administered. After a long and dark year, humanity had risen to the challenge and developed a way to combat the disease.

But, COVID-19 was not the first disease that human beings managed to surmount. The past two centuries have been rife with examples of humankind’s ability to treat—or even cure—conditions that were once considered untreatable or unpreventable.

The following examples represent some of the greatest triumphs in modern medicine’s battle against disease.

Diseases requiring antibiotics

Before the advent of antibiotics, life was hell for many whose immune systems failed to stave off infection. The saddest stories were those of kids. Bacterial meningitis killed 90% of children it infected. Those children who lived went on to experience severe, lifelong disabilities, including mental retardation and deafness. Strep throat could kill. Ear infection often beelined for the brain. Whooping cough, pneumonia, and tuberculosis tore through communities laying a path of destruction along the way.

Fortunately, things changed once Alexander Fleming serendipitously discovered and then isolated Penicillium notatum in 1928. He later collaborated with British pharmaceutical companies to produce penicillin in mass quantities. During World War II, penicillin saved the lives of countless Allied troops and was nicknamed “the wonder drug.”

But despite once being a focus of human ingenuity, the development of new antibiotics has fallen off a cliff. To address the palpable need for new antibiotics to fight antibiotic-resistant infections, a coalition of more than 20 leading pharmaceutical companies launched a fund to catalyze antibiotic development in mid-2020. The AMR Action Fund will invest nearly $1 billion in smaller biotech companies with the goal of bringing between two and four new antibiotics to market by 2030.

According to the Center for Disease Research and Policy (CIDRAP) at the University of Minnesota, “The fund is a response to the poor market conditions for new antibiotics, which compete with older, cheaper antibiotics and are used sparingly to maintain their effectiveness, resulting in poor sales. These conditions have led many large pharmaceutical companies to abandon antibiotic development altogether for more lucrative drugs, while several smaller biotech companies—which dominate the antibiotic development space—have gone bankrupt despite bringing new and novel antibiotics to market.

“As a result, the pipeline for new antibiotics is weak, with few novel candidates to address the multidrug-resistant pathogens becoming more prevalent. People involved in the effort say the money will provide a temporary boost while policymakers craft solutions to address the larger economic issues hampering antibiotic development.”

Hepatitis C

Once upon a time, hepatitis C was written off as a chronic disease with minimal treatment options. The past decade, however, has seen a seismic shift in how the virus is diagnosed and treated.

“Of the viral hepatitides, none has seen as much transformation in available pharmacologic treatment options as for chronic HCV, which has been viewed previously as a chronic infection with only modest cure rates (sustained virologic response [SVR] up to 63%) with interferon-based therapies,” wrote the authors of a review published in Hematology Communications.

“However, now it has become a disease with multiple, well-tolerated, finite therapies with SVR rates greater than 95% across nearly all patient and viral characteristics. Availability of such treatment has altered the landscape of patient populations who are able to receive treatment, as well as which patients should have access to these safe, effective treatments,” the authors added.

So what does the future look like for a viral infection with an SVR topping 95%? Current efforts are focusing on the identification of new biomarkers for the disease and the development of novel therapies targeting multiple pathways in the disease and replication pathways. To infinity and beyond!

HIV/AIDS

Most physicians vividly recall a period when HIV/AIDS lacked effective treatment. Fortunately, the federal government and others came to the rescue with the development of antiretroviral drugs that today enable those with HIV to live healthy lives.

“One of NIAID’s greatest success stories is that its research led to the development of numerous antiretroviral drugs to treat HIV/AIDS,” touts the agency, “turning what was once a uniformly fatal disease into a manageable chronic condition for many. NIAID is working to find new and more effective therapeutic products, drug classes, and combinations as well as safe and effective treatments for dangerous related co-infections and complications.”

They added that NIAID plays a role in numerous phases of the antiretroviral drug discovery and development process. “The search for new drugs remains a priority due to the development of resistance against existing drugs and the unwanted side effects associated with some current drugs. NIAID supports basic research to identify novel strategies to prevent HIV from taking hold and replicating in the body, as well as preclinical research to formulate antiretroviral drugs that can be tested in people.”

The NIAID also bolsters the biggest network of clinical trial units in the world, thus ensuring continued success in the field.

Notable advances in the field of HIV treatment and prevention include:

  • Improvement in fixed-dose combinations, such as 1-tablet-per-day options by means of fewer drugs dispatched. These alternatives also have fewer adverse effects and are longer lasting.

  • Long-acting HIV treatment options, including less frequent dose and injectable/implantable routes of administration. 

  • The emergence of pre-exposure prophylaxis (PrEP) and post-exposure prophylaxis (PEP).

  • Inroads into HIV vaccine research.

Type 1 diabetes

Before the discovery of insulin, the only way to treat type 1 diabetes was by severe carbohydrate restriction of 10 or fewer grams per day. Not only was this diet essentially torture for children, but low-carbohydrate diets [LCDs] don’t work well as treatment for this condition, according to the authors of a review published in Nutrients.

“In type 1 diabetes, there is no present evidence that an LCD or a KD [ketogenic diet] can delay or prevent the onset of the disease. These diets have the potential to improve metabolic control, but caution is needed because of the risk of DKA [diabetic ketoacidosis], of worsening the lipid profile and, in children, the unknown impact on growth,” the authors wrote.

Thank goodness for the work of a young surgeon named Frederick Banting and his assistant Charles Best who first removed insulin from a dog’s pancreas and in 1922 used it to treat a 14-year-old Canadian boy dying of diabetes. Soon thereafter, Eli Lilly mass-produced the stuff, and in 1936 Novo Nordisk Pharmaceuticals, Inc., developed a gamut of slower-acting insulins.

For many years, insulin from cattle and pigs was used, before the first genetically engineered human version of the hormone was made via E. coli. By 1982, Eli Lilly brought Humalin to market, the first commercially available biosynthetic human insulin.

According to the American Diabetes Association, “Insulin now comes in many forms, from regular human insulin identical to what the body produces on its own, to ultra-rapid and ultra-long acting insulins. Thanks to decades of research, people with diabetes can choose from a variety of formulas and ways to take their insulin based on their personal needs and lifestyles. From Humalog to Novolog and insulin pens to pumps, insulin has come a long way. It may not be a cure for diabetes, but it’s literally a lifesaver."

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