Biomedical Research Funded in 1998

Editor’s Note: CFRI funds cystic fibrosis research in two cycles every year. Last year the following researchers and their proposals were recommended for funding by the Research Advisory Committee (RAC) and approved by the Board of Directors and General Membership of CFRI. The total funds awarded for 1998 amounted to $289,114.

Spring Cycle

  1. Homer Boushey, M.D., University of California at San Francisco, Principal Investigator. Effects of Clarithromycin (Biaxin) on Sputum Production and Markers of Inflammation in Patients with Cystic Fibrosis; a pilot study.

    2. Boushey’s goal is to obtain data to guide the proposal for a multicenter trial to document whether macrolide antibiotics affect the immune response to lung infection in CF, rather than acting primarily as antibiotics that directly kill bacteria.
    Amount funded: $35,000
     

  2. Dieter Gruenert, Ph.D., University of California at San Francisco, Principal Investigator. Small Fragment Homologous Replacement (SFHR) of Genomic CFTR in Vivo.

    3. The prospect of directly correcting the mutant portion of disease-causing genes is one of the main goals of gene therapy. SFHR is an alternative method to classic gene therapy approaches that use adenoviral vectors and transfer of CFTR--DNA by liposomes. SFHR uses small fragments of DNA to replace CFTR sequences. This approach has been successful in airway epithelial cells, and other cell lines, including a nasal polyp cell line. This is follow-up funding for Dr. Gruenert who is now advancing from the cellular level to study this transfer technique in mouse nasal airways with the goal of generating transgenic animals.
    Amount funded: $30,000
     

  3. Christine Haws, Ph.D., University of California San Diego, Principal Investigator. Cell Specificity of Bicarbonate Permeation through CFTR.

In the pancreas, malfunction of CFTR impedes bicarbonate passage that is necessary for the pancreas to perform its function of neutralizing stomach acid. The application of a patch clamp technique offers hope of rapidly eliciting the mechanism.
Amount funded: $40,000

  1. John LiPuma, M.D., Allegheny University of the Health Sciences, Philadelphia, PA, Principal Investigator. Epidemiology of Burkholderia Cepacia in Northern California.

    The specific aims of this project are to a) determine the frequency with which misidentification of B. cepacia and related bacteria may be occurring in Northern California, and b) by using bacterial genotyping methodology, investigate the molecular epidemiology of B. cepacia and related bacteria in Northern California. This is a collaborative study involving the Northern California CF Centers and their laboratories.
    Amount funded: $32,883

  2. Fred Lorey, Ph.D., Chief of Program Development and Evaluation Section of Genetic Disease Branch, California State Department of Health Services to attend the Fifth International Conference on Neonatal Screening for Cystic Fibrosis in Paris, September, 1998.
    Amount funded: $1,487
  1. Stipends for research assistants studying CF in the labs of:
  • Children’s Hospital at Oakland Research Institute, Oakland, California
  • Stanford University Medical Center, Stanford, California

Amount funded: $10,746

 Fall Cycle

  1. John LiPuma, M.D., Allegheny University of the Health Sciences, Philadelphia, Pennsylvania, Principal Investigator. Gram Negative Epidemiology Studies of Seven Northern California CF Treatment Center Laboratories.

    This request is for a 6-month extension of his grant funded in the Spring cycle (see above), to include polymerase chain reaction (PCR) (genetic) as well as sputum culture tests for the presence of B. cepacia.
    Amount funded: $23,000

     
  2. Richard Moss, M.D., Director of the Cystic Fibrosis Center, Lucile Salter Packard Children’s Hospital at Stanford University Medical Center, Stanford, California. Research Nurse/Educator.

    This one-year-only grant will fund a position for a Research Nurse/Educator for the Cystic Fibrosis Center at Stanford University Medical Center to facilitate clinical research and possibly to facilitate regional collegial research.
    Amount funded: $64,000
     

  3. Neal Schiller, Ph.D., University of California at Riverside, Principal Investigator. Antibacterial Activity of Protegrin: A Novel Therapeutic Agent vs. CF.

Dr. Schiller will study a new type of antibiotic protegrin called PG-1, to determine the mechanism of action by which it kills pseudomonaes and whether this depends on the presence of certain cell surface components which may differ from the components of B. cepacia. Characterizing the bactericidal action of PG-1 against Pseudomonas aeruginosa and B. cepacia, as well as the mechanism of bacterial resistance to PG-1, are essential steps in determining the potential usefulness of PG-1 as a therapeutic agent for CF patients.
Amount funded: $37,000

  1. Student internships were given to the following laboratories with the stipulation that it be used directly for cystic fibrosis research. Total amount of awards: $14,998
  • University of California at San Francisco
  • Children’s Hospital Oakland Research Institute, California
  • Stanford University Medical Center, Stanford, California