Skip navigation

NLM Director’s Comments Transcript
The Exceptional and Cancer Medication Lessons: 06/10/2013

Picture of Dr. Lindberg

Greetings from the National Library of Medicine and MedlinePlus.gov

Regards to all our listeners!

I'm Rob Logan, Ph.D. senior staff National Library of Medicine for Donald Lindberg, M.D, the Director of the U.S. National Library of Medicine.

Here is what's new this week in MedlinePlus.listen

The National Cancer Institute (NCI) will investigate why cancer trial drugs are sometimes highly therapeutic for one or two recipients at the same time the medication fails to help a larger, intended audience, reports a news story recently published in Science.

Science explains NCI hopes the unprecedented research may be a gateway to comprehend why drugs occasionally work for just a few -- and also understand some underlying biomarkers that could boost the overall clinical efficacy of prescription cancer medications.

Science notes cancer researchers call patients ‘exceptional’ when a proposed prescription drug works for them but not for others.

Science explains cancer as well as other biomedical researchers identified and discussed the ‘exceptional’ patient phenomena for decades, but until recently did not systematically assess how the knowledge gained from the genotype of a few persons might foster tailored medications that could benefit additional patients.

The article notes NCI researchers recently found distinctive genotypes in two ‘exceptional’ cancer patients. Science explains the insights from their unusual genotypes may provide biochemical insights that might be used to modify a drug so it can be better tailored to meet the biochemistry of additional recipients.

Dr. Harold Varmus, NCI’s director, told Science (and we quote): “We want to find ways to accelerate movement towards precision medicine’ (end of quote). Varmus added cancer researchers also benefit from the insights provided by identifying unique genetic biomarkers.

In addition, NCI scientists estimate as many as four percent of persons with cancer may have genetic mutations similar to ‘exceptional’ patients. Hence, even if the identification of their genetic biomarkers does not result in a cancer drug’s improved tailoring and efficacy for most intended recipients, the number of persons who can be treated successfully may be higher than a few isolated cases.

The article explains NCI’s approach is unusual because it emphasizes understanding a medication’s effectiveness among one or two patients instead of the larger populations that usually are the focus of clinical research. While the article emphasizes NCI has no plans to abandon generalizeable clinical trial research, NCI sees single case genetic analyses as providing supplemental and complementary knowledge.

As NCI’s new research occurs, MedlinePlus.gov’s clinical trials health topic page provides a resource to help you understand how clinical trials are conducted for cancer drugs as well as other diseases and conditions.

A website from the Mayo Foundation for Medical Education and Research explains how clinical trials provide opportunities to test new therapies. This site is available in the ‘overviews’ section of MedlinePlus.gov’s clinical trials health topic page.

Some helpful information about participating in a clinical trial is provided by the U.S. Food and Drug Administration in the ‘specific conditions’ section of MedlinePlus.gov’s clinical trials health topic page.

Clinicaltrials.gov (a sister website of MedlinePlus.gov) also provides a helpful primer on the need for clinical trials as well as common research procedures.

MedlinePlus.gov’s clinical trials health topic page contains links to the latest pertinent journal research articles, which are available in the ‘journal articles’ section. From the clinical trials health topic page, you can sign up to receive email updates with links to new information as it becomes available on MedlinePlus.

To find MedlinePlus.gov’s clinical trials health topic page, just type ‘clinical trials’ in the search box at the top of MedlinePlus.gov’s home page. Then, click on ‘clinical trials (National Library of Medicine).’ MedlinePlus.gov also an array of health topic pages devoted to information about chemotherapy, the diagnosis/screening, treatment, prevention, and management of different types of cancer as well as living with cancer.

Before I go, this reminder… MedlinePlus.gov is authoritative. It's free. We do not accept advertising …and is written to help you.

To find MedlinePlus.gov, just type in 'MedlinePlus.gov' in any web browser, such as Firefox, Safari, Netscape, Chrome or Explorer. To find Mobile MedlinePlus.gov, just type 'Mobile MedlinePlus' in the same web browsers.

We encourage you to use MedlinePlus and please recommend it to your friends. MedlinePlus is available in English and Spanish. Some medical information is available in 43 other languages.

Your comments about this or any of our podcasts are always welcome. We welcome suggestions about future topics too!

Please email Dr. Lindberg anytime at: NLMDirector@nlm.nih.gov

That's NLMDirector (one word) @nlm.nih.gov

A written transcript of recent podcasts is available by typing 'Director's comments' in the search box on MedlinePlus.gov's home page.

The National Library of Medicine is one of 27 institutes and centers within the National Institutes of Health. The National Institutes of Health is part of the U.S. Department of Health and Human Services.

A disclaimer — the information presented in this program should not replace the medical advice of your physician. You should not use this information to diagnose or treat any disease without first consulting with your physician or other health care provider.

It was nice to be with you. I look forward to meeting you here next week.