Giant leap into genomic medicine

HARTFORD, Conn.—Taking a "giant leap" into the emergingfield of genomic medicine, scientists from the Jackson Laboratory (JAX), alongwith cancer specialists at Hartford Hospital and Connecticut Children's MedicalCenter, are collaborating on a three-year study to explore tailored cancertherapies for adults and children. The joint agreement is considered a majorstep toward developing more effective treatments in the ongoing war againstcancer.

Connecticut Gov. Dannel Malloy set the stage for the genomicpartnership in 2011 with his Bioscience Connecticut Initiative, resulting inthe founding in 2012 of JAX Genomic Medicine in Farmington and placing JAX inthe forefront of the emerging revolution in genome-based medical care, withemphasis on individualized therapies for cancer and other diseases.

"This partnership will advance the treatment of disease,position Connecticut as a leader in genomic medicine and impact people livingwith disease in the most meaningful way," Malloy stated in an April 30 pressrelease.

Dr. Edison T. Liu, JAX's president and CEO, underscores theimportance of establishing formal links with the "distinguished" Connecticuthospitals.

"Together, we have the potential to diagnose and treatcancer based on the unique genomic profile of the patient and the tumor," Liusays. "This collaboration will create the capability for translational genomicmedicine in Connecticut. Our goal is more effective cancer care at a lower costto patients and to society."

According to Mike Hyde, vice president of external relationsat JAX, the Connecticut connection represents just part of the company's role.

"We opened the Jackson Laboratory for Genomic Medicine inConnecticut to be a part of that state's deep commitment to the biosciences,"Hyde says. "We found strong and enthusiastic clinical partners, there, to team withour longstanding expertise in genetics and our growing mastery of computationalbiology."

JAX, a nonprofit organization, is funding the project. Anysurpluses generated by its nonprofit mouse distribution operations arereinvested in the organization's scientific research.

The cancers to be studied are those with high risk ofrelapse or recurrence, including certain types of pediatric cancer and coloncancer in adults. Over time, the study will establish a far-reaching databasethat correlates cancer treatment results with the specific genomic variationsobserved in tumors. JAX will make this information available to the globalbiomedical community.

"We are seeking clinical partners for our work in developingcancer 'avatars'—implanting human tumors into immunodeficient mice as a basisfor comparing results of various treatment options," Hyde tells DDNEWS.

Researchers will transplant and grow patient tumor tissue inthe mice, creating a cadre of patient-specific "avatars." By testing a numberof therapies in the mouse avatars and correlating the results with theparticular genetic makeup of the tumor tissue, scientists and clinicians willgain deeper understanding of the effectiveness of various therapies forspecific cancers, according to JAX.

"In effect, these are clinical trials with an 'N' of one. Wehave implanted a tumor from the first patient, as the study is underway.Eventually, we hope to use the results of such trials to advise physicians onthe best treatment options for individual patients," says Hyde.

Dr. Andrew L. Salner, director of the Helen & Harry GrayCancer Center at Hartford Hospital, says the collaborative effort allows "thefinest minds in the country to further research and win the war on cancer."

"Together, we can take the patient's own tumor cells, studytheir genomics and behavior in the lab and ultimately translate those findingsinto a personalized and effective treatment approach," Salner adds. "Doing thiswill be a giant leap forward in the development of safe and effective cancertherapies."

Dr. Fernando Ferrer, surgeon-in-chief, executive vicepresident and director of the Division of Urology at Connecticut Children'sMedical Center, says the collaboration brings with it "the hope for discoveryof new, more effective treatments for our children suffering with high-riskcancers for which effective treatments are not available. These efforts placeour institutions and our state at the forefront of a new era of cancertreatment."