PHYSICIANS’ ABILITY TO COMBAT RARE DISEASES HAMPERED BY FUNDING REDUCTIONS

EXTINCTION OF SCIENTIFIC EXPERTISE A PRODUCT OF FEDERAL BUDGET CUTS

FEATURING: Louis Weiss MD, MPH, Professor of Medicine and Pathology, Albert Einstein College of Medicine, Bronx, New York

 

STOPPING DANGEROUS INFECTIONS THROUGH RESEARCH

Opportunistic fungal pathogens, known as microsporidia, are a regular cause of disease in humans. They often cause diarrhea and wasting in immunocompromised individuals –including those with HIV, transplant patients and the elderly. While microsporidia typically infect the gastrointestinal tract, reproductive, respiratory, muscle, excretory and nervous system infections are also possible. The infections can result in a wide range of clinical disease. The organisms can be found in human water supplies and continue to emerge around the world.

Through multi-year grant support from the National Institutes of Health (NIH), Dr. Louis Weiss and his colleagues at the Albert Einstein College of Medicine have made inroads into the understanding of microsporidia. Some of the successes of Dr. Weiss’ research team include the identification of new infections caused by microsporidia; establishing diagnostic primers for the molecular diagnosis of microsporidia, defining the composition of the invasion structure used by these organisms to infect host cells; identifying a therapeutic target and new compounds that inhibit this target for use in the therapy of microsporidiosis; and helping to develop a consortium that sequenced the genomes of many of the species of microsporidia that infect humans. 

CUTS LEAVE UNANSWERED QUESTIONS

Their work has been supported by roughly $250,000 per year through the NIH’s National Institute of Allergy and Infectious Diseases (NIAID), AIDS-associated Opportunistic Infections and Cancer grant program.  However, over the last few years, as the NIH and NIAID budgets failed to keep pace with inflation and indiscriminate federal austerity measures such as sequestration began to further impede biomedical research, Dr. Weiss’ grant was cut.  

Personnel with considerable expertise were lost from Dr. Weiss’ team. Several lines of inquiry on microsporidia were halted as the NIH pulled back its support. Researchers are still uncertain as to how infection occurs.  It’s also unclear how pathogens cross species, such as from insects to humans.   Researchers lack genetic systems to allow us to better understand these pathogens.  For instance, currently microsporidia can’t be genetically manipulated to understand how they work or to make them less virulent.  There are no drugs that are effective against all of the species that infect humans.  Much of the NIH investment in this area over 20 years will fail to yield the lifesaving results that seemed possible just a few years ago.

Sequestration has helped move NIH’s grant approval line from the twentieth percentile for many institutes and centers to the single digits, which means that promising science is left on the table that could otherwise be funded. The decline in support has been acute for studies of rare pathogens, which threatens to diminish scientific diversity.  According to an article by Dr. Weiss and colleagues: 

There is a real and present danger that extinction of expertise may be occurring due to changes and fluctuations in funding.  The limitations of working on difficult systems or rare diseases are that they may be superficially viewed as less significant or important… loss of such expertise, which is already limited by the relatively small size of research communities, could promote the extinction of critical resources.  

The research undertaken by Dr. Weiss’ laboratory has already directly impacted patients. His laboratory has been involved in many cases where they were able to make a correct diagnosis and to help with drug therapy. In many of these cases pathogens were either initially missed or incorrectly identified.  Dr. Weiss’ team is left to contemplate what tests and cures might have materialized if NIH had the funding to carry this research forward. 


AUTHOR
Infectious Diseases Society of America

SOURCES
i. Eukaryot Cell. 2012 Feb; 11(2): 90–97. doi: 10.1128/EC.05143-11.