MMRC tissue bank

Patients With Rare Diseases Work to Jump-Start Research

Advocacy Groups Create Their Own Tissue Banks To Aid in Drug Development

By AMY DOCKSER MARCUS

Kathy Giusti, 47 years old, was diagnosed a decade ago with multiple myeloma, an incurable and rare cancer of the blood. With only 16,000 new cases diagnosed a year, Ms. Giusti, a former pharmaceutical-company executive, knew that it would be hard to get drug companies and researchers to study her disease.

When she asked researchers at a 2004 meeting what it would take to speed up the search for treatments, she says they told her that "the biggest obstacle was getting tissue from multiple myeloma patients to study and test." So she decided to give them something to get started: In 2005, the Multiple Myeloma Research Consortium, founded by Ms. Giusti, started its own biospecimen bank.

With more than 600 samples of bone marrow and blood already collected from around the country, the bank's resources are being used to support important new research. The consortium, which now includes 11 academic institutions, earlier this year launched a Multiple Myeloma Genomic Initiative, which will study the tissue samples to look for genes that play a role in the disease. It is also discussing with researchers in France the possibility of setting up a European-based multiple-myeloma bank to further expand the supply of tissue.

Biospecimen banks, which may include tissue samples, blood, spinal fluid or other specimens along with clinical data about patients, are critical to medical research. They offer researchers a way to test new drugs and to study and better understand the biology of a disease. And they offer the best way of finding new targets for experimental drugs.

Major drug companies and medical centers often maintain such banks for a range of diseases. But specimens sometimes sit unused because there isn't money to study them. If a disease is rare, individual centers may not see enough patients to collect a critical mass of samples, and smaller drug companies often don't have the resources to find patients. Patient groups have also found that study results often aren't shared with other researchers, and legal fights have broken out over who owns the rights to potential therapies developed from tissue.

Out of frustration over all these issues, patient groups—especially ones focused on rare, underfunded diseases—are increasingly taking matters into their own hands. They are starting their own biospecimen banks in the hopes of accelerating research and gaining more control over the process.

Ms. Giusti says it cost $2 million to $3 million to set up the multiple-myeloma bank, paid for partly by a grant from the Pioneer Fund, a private family foundation in Denver. Maintaining it costs about $1.5 million a year, which goes mainly to salaries for assistants at major cancer centers who oversee the collection of specimens, the cost of collecting and shipping them, and storage at the Mayo Clinic Scottsdale, in Arizona, where the bank is housed.

At a meeting of patient-advocacy groups last month in Cambridge, Mass., Ms. Giusti recounted some of the challenges she faced in launching the tissue bank. Often, even at major academic centers, the bank wasn't able to get large numbers of samples, so organizers decided they needed more centers. Ms. Giusti estimated that it cost her group $250,000 in legal fees to get a membership agreement written and signed by all the centers.

The Multiple Myeloma Research Consortium has developed stringent standards of its own for collection and storage of samples. At sites around the country that work with the consortium, anytime a patient with multiple myeloma has a bone-marrow draw, he or she is asked to donate an extra sample for the bank. The samples are all drawn, bar-coded, shipped and treated according to a protocol developed and paid for by the consortium. Every week, the consortium sends out a kind of "report card" to each center which tells them how many samples each site has shipped. A system was set up to allow each center to track the specimens and enter patient data in a standard fashion.

The Mayo Clinic storage facility is neutral on how the specimens are used, says Rafael Fonseca, associate professor at the Mayo Clinic and medical director of the tissue bank; such decisions are vetted by the consortium's steering committee.

The multiple myeloma tissue bank has played a key role in several new projects. Nereus Pharmaceuticals of San Francisco plans to start a clinical trial with a new drug, and the tissue bank will collect samples from the patients treated with the drug to allow the company to determine if the drug is working and which patients are responding. Novartis is testing an experimental drug in a group of patients who have an aggressive subtype of the disease that hasn't responded well to standard treatments, and will use tissue from the bank to identify patients in that group.

Source: excerpted from “Wall Street Journal online”