: We propose to develop a clinical non-viral therapy for treatment-induced anemia in AIDS/HIV and chemotherapy patients using a proprietary TriGrid electroporation system (EPT). Anemia is currently treated with repeated administration of recombinant erythropoietin (EPO), which is expensive and not readily available to these patients due to the already elevated cost of treatment for their primary disease. However, correction of this anemia will not only improve quality of life, but may also have the potential to improve these patients' probability of survival. Thus, development of a more cost effective and equally efficacious method of EPO therapy such as EPT-delivery of an Epo plasmid, is clearly justified.Ichor's Phase I work has shown the feasibility of delivering DNA plasmids containing genes such as erythropoietin (Epo), into skeletal muscle of small and large rodents using EPT. Phase II work will involve a comprehensive dose response analysis, and characterization of treatment toxicity and vector biodistribution. Using a clinically relevant plasmid construct, initial experiments will include testing in normal and diseased rodent models to evaluate biologic response and establish efficacy and dosing. A large animal study will be conducted to verify dose scale up and identify potential toxicity and safety issues associated with the therapy.

Proposed Commercial Applications

The estimated sales of Epogen and Procrit, recombinant forms of erythropoietin (EPO), are above $2 billion annually. The development of a treatment which allows the body to manufacture its own EPO to stimulate red blood cell production in response to treatment- induced anemia, is very enticiting. By introducing and expressing the EPO gene in muscle using Ichor's electroporation technology, the body would be able to produce EPO for a more extended amount of time and would significantly decrease the number of treatments patients would require to treat their anemia, thus decreasing the overall cost of treatment. The already high cost of care for AIDS and cancer patients often makes this treatment to improve their quality of life and perhaps their likelihood for survival, unaffordable.

Agency
National Institute of Health (NIH)
Institute
National Heart, Lung, and Blood Institute (NHLBI)
Type
Small Business Innovation Research Grants (SBIR) - Phase II (R44)
Project #
5R44HL065164-03
Application #
6527563
Study Section
Special Emphasis Panel (ZRG1-AARR (10))
Program Officer
Qasba, Pankaj
Project Start
2000-07-11
Project End
2005-08-31
Budget Start
2002-09-01
Budget End
2005-08-31
Support Year
3
Fiscal Year
2002
Total Cost
$501,842
Indirect Cost
Name
Ichor Medical Systems, Inc.
Department
Type
DUNS #
City
San Diego
State
CA
Country
United States
Zip Code
92121