Favrille, a San Diego-based biopharmaceutical company, is one of a handful of firms on the forefront of personalized medicine.
Because personalized treatment is tailored to an individual's biology, it has the potential to be far more effective than
current approaches to disease management.
Favrille focuses on developing and commercializing patient-specific therapies for immune system diseases. Its lead drug candidate
FavId, is an active immunotherapy for the treatment of B-cell non-Hodgkin's lymphoma. The company has completed patient enrollment
in a pivotal Phase 3 trial and plans to unblind the study during the first half of 2008.
In the following interview, EJ Brandreth, the senior director of quality at Favrille, talks about potential new cancer treatments,
regulatory challenges, and the wonder of disposables.
Where do we stand in the evolution of personalized medicine?
Personal medicine has been in development since the late 1980s. No one yet has a personalized product approved for the treatment
of cancer, but four or five companies are near the end of Phase 3 trials, and over the next several years, we expect to see
approvals come through. There's a big difference between personalized treatment, and personalized products. Personalized treatment
is where the doctor prescribes the best treatment for you based on your genetic profile; personalized products involve a drug
that is actually made just for you and you alone.
What has been the biggest breakthrough leading to personalized medicine?
By far, it has been the ability to rapidly sequence a patient's DNA, identify the sequences of interest, and produce recombinant
protein at a scale appropriate to the need. Over the last five to 10 years, we learned how to go into a tumor and analyze
a sample from a patient. Pathologists and doctors are now able to look at the DNA sequences (biomarkers) of cancer patients,
which tell them what the best treatments would be for the patient. This information directs the physician on which general
therapeutic can be used. If personalized products are not available yet, doctors can identify which general therapy—chemotherapy,
antibodies, or radiation—would be the most statistically effective for a particular cancer type, based on their genetic screening.
What is Favrille's current focus?
Our lead product candidate, FavId, is specific to a patient's cancer—B-cell non-Hodgkin's lymphoma. This is a cancer of the
lymphoid system and the tumor cells produce an antibody that is specific to the patient and to the tumor. Due to the advancements
in high-throughput sequencing, sequence databases and plasmid generation, and expression in an insect cell system, it is possible
to generate large amounts of the specific antibody produced by those cancer cells. These antibodies are then given back to
the patient, so his or her immune system mounts a defense against those antibodies, and therefore against that tumor. This
type of treatment should also work on other immune-system cancers.
QA: How much difference is there between the antibodies of one patient and the antibodies of another?
While all antibodies have a common structure involving two heavy and two light chains, there are variable regions that are
unique to each patient's lymphoma tumor. The probability of identical sequences in any two lymphomas is extremely unlikely.
In addition to DNA sequencing, what other developments have led to these advancements?
In addition to DNA sequencing, the use of expression systems that do not require cell transformation has been a major advancement,
such as the use of baculovirus in an insect cell culture. This dramatically reduces the time in cell processing for the production
of recombinant proteins.