Lani
07-10-2006, 10:05 PM
At a lecture I went to three months ago at Stanford, the country's leading researcher on breast cancer stem cells told me the most important thing that her2neu breast cancer patients could do would be to use their advocacy strength to try to alter the privacy policies which make it almost impossible for researchers to utilize patients' breast cancer samples in their research.
I think I mentioned this once before to Cynthia, who was looking for a way to utilize her legal connections and skills to promote a cure.
Here is another way around the problem, which is probably more effective and requires no change in the law--volunteering to give breast cancer specimens for use in research. It is problematic, as it cannot be used in double-blind type studies, as there will be a a possibility of some bias in sampling due to the altruistism and intelligence of those providing samples skewing the results in some way.
Here is the article from the Wall Street Journal:
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
July 11, 2006; Page D1
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.
Kathy Giusti, founder of the Multiple Myeloma Research Consortium, helped create a biospecimen bank for 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.
Groups that include the Accelerated Cure Project for Multiple Sclerosis, the Joubert Syndrome Foundation, Cure Autism Now and the Inflammatory Breast Cancer Research Foundation have all started banks, leveraging their extensive patient databases and ability to raise funds. The Lance Armstrong Foundation recently awarded a grant to the Keck School of Medicine of the University of Southern California to establish the Los Angeles County Germ Cell Tumor and Tissue Bank Resource, which will collect, store and distribute testicular-cancer tissue specimens for cancer researchers all over the country in the hopes of finding new drug targets for that rare cancer.
Starting a bank "is becoming an obligate strategy for patient advocacy groups if you want to make really rapid progress, especially in a rare disease," says Jeffrey Trent, the president and scientific director of the Translational Genomics Research Institute, which along with the Eli and Edythe L. Broad Institute of MIT and Harvard, is working on the Multiple Myeloma Genomic Initiative. "We could not do this genomic project on multiple myeloma without the existence of the biospecimen bank."
Running a bank can be an expensive proposition. The Accelerated Cure Project for MS Repository says it will cost $2.5 million to collect the first 1,000 samples of blood and clinical data from people with multiple sclerosis and matched controls. The Genetic Alliance Biobank, established by seven patient-advocacy groups in 2004 as a way of keeping down expenses and standardizing operating procedures, says it costs advocacy groups $13,000 a year to join and use the alliance biobank.
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 surge in patient-run banks comes at a time of controversy and discussion about the future of banks in cancer research. In 2002, the National Dialogue on Cancer, a forum of private-sector, academic, government and nonprofit groups, identified access to high-quality tissue as a key barrier in drug development.
There are no national standards for repositories that collect and store the specimens used in cancer research, or even consensus on what are the necessary storage conditions for maintaining the tissue. The prospect of patient groups starting banks has exacerbated concerns about material not being properly collected or stored "and having 1,000 advocacy groups just doing their own thing," says Paula Kim, president and founder of Translating Research Across Communities, which is working on the issue of creating a national network of biospecimen banks.
After much study, the National Cancer Institute's Office of Biorepositories and Biospecimen Research just released guidelines to try to standardize the policies at the 125 NCI-supported banks. A pilot project in which eight NCI-sponsored prostate-cancer centers store and exchange tissue using standard procedures started a year ago. The hope is that independent biobanks will adopt the same standards.
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.
For many patient groups, frustrated by the dearth of treatment options, running a bank gives them the power to shape and drive forward the search for treatments. Sharon Terry, who has two children with a rare genetic disease called pseudoxanthoma elasticum and is the founding president of the Genetic Alliance Biobank, says, "We have 33 scientists working under my coordination, largely because I hold the resource, the blood and tissue bank."
Write to Amy Dockser Marcus at amy.marcus@wsj.com
I think I mentioned this once before to Cynthia, who was looking for a way to utilize her legal connections and skills to promote a cure.
Here is another way around the problem, which is probably more effective and requires no change in the law--volunteering to give breast cancer specimens for use in research. It is problematic, as it cannot be used in double-blind type studies, as there will be a a possibility of some bias in sampling due to the altruistism and intelligence of those providing samples skewing the results in some way.
Here is the article from the Wall Street Journal:
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
July 11, 2006; Page D1
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.
Kathy Giusti, founder of the Multiple Myeloma Research Consortium, helped create a biospecimen bank for 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.
Groups that include the Accelerated Cure Project for Multiple Sclerosis, the Joubert Syndrome Foundation, Cure Autism Now and the Inflammatory Breast Cancer Research Foundation have all started banks, leveraging their extensive patient databases and ability to raise funds. The Lance Armstrong Foundation recently awarded a grant to the Keck School of Medicine of the University of Southern California to establish the Los Angeles County Germ Cell Tumor and Tissue Bank Resource, which will collect, store and distribute testicular-cancer tissue specimens for cancer researchers all over the country in the hopes of finding new drug targets for that rare cancer.
Starting a bank "is becoming an obligate strategy for patient advocacy groups if you want to make really rapid progress, especially in a rare disease," says Jeffrey Trent, the president and scientific director of the Translational Genomics Research Institute, which along with the Eli and Edythe L. Broad Institute of MIT and Harvard, is working on the Multiple Myeloma Genomic Initiative. "We could not do this genomic project on multiple myeloma without the existence of the biospecimen bank."
Running a bank can be an expensive proposition. The Accelerated Cure Project for MS Repository says it will cost $2.5 million to collect the first 1,000 samples of blood and clinical data from people with multiple sclerosis and matched controls. The Genetic Alliance Biobank, established by seven patient-advocacy groups in 2004 as a way of keeping down expenses and standardizing operating procedures, says it costs advocacy groups $13,000 a year to join and use the alliance biobank.
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 surge in patient-run banks comes at a time of controversy and discussion about the future of banks in cancer research. In 2002, the National Dialogue on Cancer, a forum of private-sector, academic, government and nonprofit groups, identified access to high-quality tissue as a key barrier in drug development.
There are no national standards for repositories that collect and store the specimens used in cancer research, or even consensus on what are the necessary storage conditions for maintaining the tissue. The prospect of patient groups starting banks has exacerbated concerns about material not being properly collected or stored "and having 1,000 advocacy groups just doing their own thing," says Paula Kim, president and founder of Translating Research Across Communities, which is working on the issue of creating a national network of biospecimen banks.
After much study, the National Cancer Institute's Office of Biorepositories and Biospecimen Research just released guidelines to try to standardize the policies at the 125 NCI-supported banks. A pilot project in which eight NCI-sponsored prostate-cancer centers store and exchange tissue using standard procedures started a year ago. The hope is that independent biobanks will adopt the same standards.
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.
For many patient groups, frustrated by the dearth of treatment options, running a bank gives them the power to shape and drive forward the search for treatments. Sharon Terry, who has two children with a rare genetic disease called pseudoxanthoma elasticum and is the founding president of the Genetic Alliance Biobank, says, "We have 33 scientists working under my coordination, largely because I hold the resource, the blood and tissue bank."
Write to Amy Dockser Marcus at amy.marcus@wsj.com