by Tanner Godfrey
During this Senate Labor, Health and Human Services (LHHS) hearing, the focus was on the President’s FY2018 proposed budget cut of $7 billion to the National Institutes of Health (NIH). Recently reappointed director Dr. Francis Collins was the witness. I’ve previously described the commitment of the Senate LHHS subcommittee to preserve funding for NIH, so in this post I want to focus on some key points raised by Dr. Collins. In his testimony, he described three moving examples of the transformational power of investing in NIH basic science.
Cystic Fibrosis – Cryo Electron Microscopy (Cryo-EM), a powerful magnification technique, has enabled the discovery of the precise molecular structure of the protein channel that regulates water and salt balance. This protein is misfolded in cystic fibrosis (CF), our nation’s most common fatal genetic disease. Dr. Collins discovered the genetic mutation, or misspelling, responsible for this disease. Now, structural information is allowing the design of better drugs to treat cystic fibrosis patients like 2-year-old Avalin Mahoney.
“Just a few decades ago she probably wouldn’t have made it beyond her teens; no longer! Today we have two targeted drugs for CF, and more to come, all building on NIH-supported basic research. And we’re not done; the goal is to turn CF into a 100% curable disease,” Dr. Collins said.
Among the next generation of scientists tackling the problem is Dr. Steven Aller from the University of Alabama. He uses computer science and biomedical science to better design targeted drugs for such diseases.
Sickle Cell Disease – Sickle cell is a disease in which red blood cells are sickle shaped due to a genetic misspelling. These misshapen cells can block small arteries. Currently, the only cure for the disease is a bone marrow transplant. However, what of the many patients without a match? That is where current research using CRISPR gene editing by NIH’s Dr. Courtney Fitzhugh comes into play. She is attempting to correct the genetic misspelling associated with sickle cell disease in a patient’s own blood stem cells.
Alzheimer’s Disease – The NIH BRAIN Initiative aims to create a detailed understanding of the 86 billion neurons in the human brain. Using the BRAIN Initiative, scientists hope to identify early warning signs of Alzheimer’s disease, which is estimated to cost $259 billion this year, rising to over $1.1 trillion by 2050. Using advanced warning, effective means of preventing this disease. Current research is tracking a family with inherited Alzheimer’s living in the mountains of Columbia. Using PET scans of the brain they compare those who carry mutations and those who do not in hopes of identifying early warning signs of Alzheimer’s.
Dr. Collins concluded his testimony by telling the senators, “You are all a part of this, your emphasis on Alzheimer’s and related dementias research in fiscal 2016 & 2017 is enabling progress towards our mutual goal of preventing and effectively treating these devastating conditions.”
While taking questions from committee members, Dr. Collins again addressed the issue of capping indirect costs. He said the NIH will look for ways to reduce administrative costs brought on by regulations, but he does not think it will amount to the proposed dramatic rate reduction. Asked about the effect of capping indirect costs at the proposed 10% rate, Dr. Collins replied, “I’m having a hard time imagining how we would manage that.”