Basic Science, Clinical Research, and Kidney Disease Treatments

When you hear the word “research,” you may think about innovative treatments and breakthrough technologies but these cutting-edge therapies are only made possible with another type of research called basic science. What is basic science research, and how does it apply to kidney disease? Dr. Holly Kramer, Dr. Joseph V. Bonventre, and Ph.D. candidate Hannah Wesselman break it down. 

What is basic science?

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Basic science studies how the body works and how diseases change it over time.

"In regards to kidney disease, basic science explains how the kidneys filter blood and excrete waste products. It tells us that the kidney is involved in maintaining blood pressure, how it regulates components of the blood, and how it balances fluids in the body. It tells us how kidneys make hormones, which affect the bone and blood content of the body," said Dr. Bonventre. "Basic science also explains what can go wrong with the kidneys."

To study the kidneys and other human body systems, scientists use animal models.

"I work with zebrafish as a model organism to study kidney development. While the zebrafish's kidney looks different from a human, the basic fundamental processes are similar," Wesselman said. "We can use this model to ask questions that are important to answer in order for us to get to more applicable human studies."

For example, one area of research involves identifying every cell in the kidney.

"We want to know what the cell does under normal conditions, how it changes in response to disease, and how they interact with each other during both conditions," said Dr. Bonventre. "We’re trying to understand how nature puts everything together. By taking it one piece at a time, we can generate part of it. Someday we hope to generate an entire new kidney in a dish so we can give it to patients in need."

Want to get involved in kidney disease research? Sign up to find current patient-centered research studies looking for people like you to share their insights and experiences.

Bridging the gap between theory and application

Understanding the kidney's structure and how it works is only the start. Researchers also use basic science to determine whether or not new treatments cause more harm than good.

"You can test organisms like zebrafish to see if something works to protect the kidney or is toxic to it. This is very important because many drugs developed for patients with problems like cancer or heart disease are excreted or metabolized by the kidneys and liver," said Dr. Bonventre. "This type of testing allows us to develop or use these new treatments in vitro, meaning outside of the body. Afterwards, we can predict whether or not we need to worry about the patient's kidneys when they're given these drugs."

These foundational studies also allow scientists to test medications or therapies in people who experience issues like low blood pressure.

"We can make the animal model's kidney have less oxygen to test what happens if someone's blood pressure goes very low. We can test what would happen if a patient develops an infection during surgery," Dr. Bonventre said. "We treat the animals with a variety of drugs to see which works. We can also grow human kidney cells in a dish to see how these treatments work on human tissue before moving on to human testing."

Other scientists don't just use animals for testing purposes–some are trying to figure out how to transplant an animal's organ to the human body, called xenotransplantation.

"There aren't enough human organs to go around," Dr. Bonventre said. "Pig kidneys could help more people receive timely transplants."

Learn about the recent xenotransplant breakthroughs.

Moving to clinical research

Once animal testing is complete, scientists move to clinical research. 

"Clinical research is a broad term. It could mean utilizing previously collected data or doing cohort studies, where scientists follow people over time to see who does or does not develop disease and how a disease progresses," Dr. Kramer said. "Clinical trials, where people are randomly assigned an intervention or a placebo, also fall into the clinical research category."

Clinical trials have different stages, each with its own goals. If a new medication or treatment is successful in one stage, it moves on to the next until it gets approved by the FDA for public use. Even after approval, more testing is done to learn about its effects on people with different diseases or in the long term.

The phases of clinical trials include:

• Phase I: Testing a small group to find the best dosage and identify any side effects.
• Phase II: Testing involves more people to see if the treatment works and if most people can tolerate it.
• Phase III: Compares the new treatment with existing ones to see if it's better or has fewer side effects.
• Phase IV: The treatment is approved for public use, and further trials are conducted to see how it affects specific groups, like those with kidney disease.

Learn if clinical trials are for you.

Funding new research

Despite the prevalence of chronic kidney disease, kidney disease research doesn't receive nearly the same amount of financing as other chronic diseases. 

In 2020, the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) spent only $700 million on chronic kidney disease research, just 2% of the funding provided by the National Institutes of Health (NIH). Funding for the NIH increased by 37% between 2015 and 2020, but the budget for kidney research only increased by about half of that percentage.

Learn more about kidney disease funding.

"We're only spending about $18.00 per patient for kidney research compared to $305 per cancer patient and $50 per cardiovascular patient. We need money for the basic and clinical sides of research to make significant progress," Dr. Kramer said. "If you're a patient or a patient family member you can help by joining NKF's advocacy group, Voices for Kidney Health. We need your voice to get people to recognize that this largely asymptomatic disease is killing millions every year and reducing quality of life."

Become a Voices for Kidney Health advocate and tell your federal lawmaker to support our funding efforts now.

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