Seattle BioMed Initiates Phase 1 Safety Studies with its Malaria Vaccine Candidate

Date: 
Thu, 05/13/2010

Marks Milestone for Infectious Disease Research-Focused Non-Profit Organization

SEATTLE, May 13, 2010 –Seattle Biomedical Research Institute (Seattle BioMed) announced today the initiation of a Phase 1 clinical safety trial – the first test in human volunteers – of its investigational genetically engineered whole parasite malaria vaccine candidate. This approach to vaccine development – using a weakened form of the whole organism that causes a particular disease – has proven successful in eradicating smallpox, as well as controlling diseases such as flu and polio but has never been used successfully for fighting human parasites. Funding for this research project comes from the Foundation for the National Institutes of Health through the Grand Challenges in Global Health initiative.

“At Seattle BioMed, we know that our ultimate success will be measured in the number of lives saved by our discoveries,” said Ken Stuart, president and founder of Seattle BioMed. “With this malaria vaccine safety trial, our progress will be measured by new knowledge we gain in the battle against malaria.” Stuart added that this trial marks an important milestone for Seattle BioMed as an organization. “For years, we’ve been known for our groundbreaking infectious disease research,” he said. “Now we are moving our research from discovery into clinical trials.”

“Creating an effective vaccine for a parasitic disease like malaria is a complex scientific challenge,” said Dr. Tachi Yamada, president of Global Health at the Bill & Melinda Gates Foundation.  “Seattle BioMed and its partners have shown that progress is possible when innovative science is given the opportunity to succeed.”

For centuries, malaria has been one of the greatest health challenges in the world, killing about one million people annually and destroying – through premature death and disability – the equivalent of at least 35 million years of healthy, productive human life each year. While other malaria vaccine candidates are being tested, Seattle BioMed’s vaccine is unique in two aspects: the use of a specifically weakened form of the whole organism and the use of gene knock-outs in the parasite.

“The use of the whole parasite as a vaccine – rather than a single building block of the parasite – stimulates the immune system in the broadest way, providing the best protection against malaria,” explained Stefan Kappe, Ph.D., a principal investigator at Seattle BioMed, who developed the vaccine in partnership with colleagues at the Walter Reed Army Institute of Research (WRAIR) and the Walter and Eliza Hall Institute of Medical Research in Australia. “And, through precise deletion of two key genes in the parasite, we have made a live vaccine that gives complete protection in mouse studies.”

Having met U.S. Food and Drug Administration’s (FDA) requirements, Seattle BioMed’s first malaria vaccine candidate will be tested for safety in healthy U.S. volunteers. The trial began this week at WRAIR, which has a long-standing history of safely testing malaria vaccine candidates. If the vaccine is shown to be safe and well-tolerated, a Phase 2 trial, planned for late 2010 or early 2011, will investigate the vaccine’s efficacy.

 “Our highest priority is to determine if this type of live-attenuated vaccine is safe in humans,” said Kappe. “We anticipate achieving protection, but the level of protection is hard to predict. Though we hope this vaccine candidate will prove safe and highly protective, we are already making significant advances in the development of next-generation attenuated vaccine strains as part of our clinical development plan.”

Kappe’s vaccine research is focused on the initial stage malaria infection when the parasite is transmitted by a mosquito bite and infects the liver but does not yet cause disease. It is only later when the parasite spreads to the blood stream that it can cause severe disease and death.

The genetically attenuated parasite (GAP) vaccine candidate is created by deletions of two genes that are predicted to stop the P. falciparum parasite – which causes the most deadly form of human malaria – during liver infection and block it from multiplying in this stage. However, the GAP is still alive and should be able to stimulate the body’s protective immune system to recognize and destroy incoming infectious parasites. “Because the genes are deleted from the genome, the parasite cannot recreate them,” Kappe explained. “In addition, the ‘one-two punch’ approach of deleting two essential genes is designed to prevent the attenuated parasite vaccine from restoring its capacity to multiply and lead to blood infection. This safety trial is just the beginning of our endeavor to develop a safe and protective malaria vaccine.”

Grand Challenges in Global Health
Seattle BioMed’s vaccine has moved rapidly from the discovery phase to the translation phase, in keeping with the intent of the Grand Challenges in Global Health initiative, which provided funds for the vaccine’s move “from mice to men.”  The Grand Challenges initiative was launched by the Bill & Melinda Gates Foundation in 2003. The initial round of projects started in 2005 were supported by $450 million from the Gates Foundation, $27.1 million from the Wellcome Trust, and $4.5 million from the Canadian Institutes of Health Research (CIHR) including a $200 million commitment managed by the Foundation for the National Institutes of Health to fund and manage a number of the projects.
 
In 2005, Kappe was one of 43 researchers funded at the initial launch from over 1,500 proposals submitted from 75 countries. All of the Grand Challenge projects seek to tackle one of 14 major scientific challenges that, if solved, could lead to important advances in preventing, treating, and curing diseases of the developing world. Kappe’s project addresses the Grand Challenge goal of creating new vaccines.

The Grand Challenges “fast track” approach is targeted at utilizing the best science and technologies to accelerate more groundbreaking advances in global health over the next decade than in the past 50 years.

ABOUT SEATTLE BIOMEDICAL RESEARCH INSTITUTE:
Seattle BioMed is the largest independent, non-profit organization in the U.S. focused solely on infectious disease research. Our research is the foundation for new drugs, vaccines and diagnostics that benefit those who need our help most: the 14 million who will otherwise die each year from infectious diseases, including malaria, HIV/AIDS and tuberculosis. Founded in 1976, Seattle BioMed has nearly 350 staff members. By partnering with key collaborators around the globe, we ensure that our discoveries will save lives sooner. For more information, visit www.seattlebiomed.org.

ABOUT THE FOUNDATION FOR NIH:
The Foundation for NIH was established by the United States Congress to support the mission of the National Institutes of Health – improving health through scientific discovery in the search for cures. It expedites or facilitates new discoveries by convening innovative public/private collaborative partnerships that leverage the diverse strengths of stakeholders including those from the scientific and research community, industry, academia, advocacy groups, foundations and philanthropy. The foundation is a non-profit, 501(c)(3) corporation, that raises private-sector funds for a broad portfolio of programs that complement and enhance NIH priorities and activities. http://www.fnih.org.

For more information, contact:

Lee Schoentrup, Communications Director
206.256.7440 lee.schoentrup@seattlebiomed.org

Jennifer Mortensen, Communications Specialist
206.256.7220   jennifer.mortensen@seattlebiomed.org