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Foundation News Updates
Tarlov Cyst Research Information
Spring 2009
Tarlov cyst researcher, Anne Louise Oaklander, M.D., PhD., has shared the following initial research project goals.
Tarlov Cyst Research: Specific Goals
· Identify patients with Tarlov cysts utilizing large patient databases
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Test several hypotheses including gender association, parity, connective tissue disorders, history of back injury/trauma, and infection by viruses that damage spinal sensory ganglia
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Investigate whether specific radiological features of Tarlov cysts correlate with the symptoms present. (Outcome variables will comprise cyst location, maximum visualized diameter, absence or presence of free communication with cerebrospinal fluid (CSF) as determined from flow study or comparing T2 MRI signal in cyst fluid and CSF).
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Measure correlations between radiological variables and the presence of neurogenic bladder or bowel, chronic leg pain paresthesias, chronic pelvic or perineal pain, low back pain,(and sensory dermatomes
February 28, 2009 was Rare Disease Day in the United States and Europe
This February 28, 2009, thousands of patients, their families, medical professionals, researchers, educators and others will join in the observance of the first Rare Disease Day in the United States.
Governors in Alabama, Connecticut, Florida, Georgia, Iowa, Kentucky, Louisiana, Maine, Maryland, Massachusetts, Michigan, Missouri, Montana, Nebraska, New Hampshire, New York, Oklahoma, Oregon, Pennsylvania, South Carolina,South Dakota, Tennessee, Texas, Virginia and West Virginia issued Proclamations that February 28, 2009 is Rare Disease Day. This day is an opportunity to increase awareness about rare diseases and the difficulties encountered by those who are diagnosed with a rare disease.
Rare Disease Day was observed for the first time February 29, 2008 in Europe and was a great success. This year, more than 100 rare disease organizations in the United States are partnering with the National Organization of Rare Disorders (NORD) and with the European Organization for Rare Diseases (EUORDIS) to improve awareness of rare diseases throughout the U.S. and Europe.
In the U.S., a rare disease is defined as one that affects fewer than 200,000 Americans. Yet since there are nearly 7,000 such diseases, approximately 30 million Americans suffer from a rare disease, according to the National Institutes of Health (NIH).
People with rare diseases experience certain challenges as a result of the fact that their diseases are so rare. These include:
· Difficulty in obtaining a timely, accurate diagnosis
· Limited treatment options
· Difficulty in finding physicians or treatment centers with knowledge and expertise
· Treatments that are often more expensive than those for common diseases
· Reimbursement issues related to private insurance, Medicare and Medicaid
· A sense of isolation and hopelessness
Rare diseases can affect the entire family of an individual patient. Caregivers often endure ongoing stress and isolation managing the medical and financial issues that arise.
One example of a rare disease that most physicians and the public have never heard of is Tarlov cyst disease. Tarlov cysts (TC) are also known as perineural/perineurial, or sacral nerve root cysts. They are spinal fluid filled cysts that form off the nerve root sheaths and can cause intense nerve pain, since the cyst walls contain spinal cord nerves. They are located most prevalently in the sacral section of the spine between the lumbar area and tailbone (coccyx). The most common symptoms that patients with Tarlov cysts experience are pain in the lower back and in the buttocks, legs and feet, weakness and/or cramping in legs and feet, abnormal sensations in the legs and feet (paresthesias), pain sitting or standing for even short periods of time, bowel and/or bladder dysfunction, headaches (due to the changes in the spinal fluid pressure), sciatica, and vaginal, scrotal, rectal, pelvic and abdominal pain. When a patient describes this group of symptoms, a lumbar-sacral scan should be ordered to rule out the presence of Tarlov cysts.
Tarlov cyst disease is often misdiagnosed/undiagnosed by the medical community, due to lack of education or understanding about the pathology of symptomatic Tarlov cysts, failure to order MRI of the sacrum, and because of the many other body systems that can be affected by the spinal cord nerves. Only a handful of physicians in the world are knowledgeable about and willing to treat Tarlov cysts. To learn more about Tarlov cyst disease, please visit the Tarlov Cyst Disease Foundation’s website at www.tarlovcystfoundation.org. The Foundation provided funding for Tarlov cyst research this year, and Anne Louise Oaklander, M.D., Ph.D., at Harvard Medical School and Massachusetts General Hospital in Boston, has received the initial research grant.
Other important things to remember about rare diseases are:
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Many rare diseases are very serious, life altering or life-threatening.
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Most rare diseases have no effective treatment or cure.
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Since many rare diseases are genetic, about half of the people affected by rare diseases in the U.S. are children.
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Research on rare diseases often adds significantly to the general understanding of more common diseases.
Research Announcement
November 2008
The National Organization for Rare Disorders’ Medical Advisory Committee has announced that Anne Louise Oaklander, M.D., Ph.D will receive the Tarlov Cyst Disease Foundation’s grant for Tarlov cyst research.
Dr. Oaklander is an attending physician in Neurology and Neuropathology at the Massachusetts General Hospital and Associate Professor of Neurology at Harvard Medical School. She directs the Nerve Injury Unit, which she founded in 2002 at Massachusetts General Hospital, following her fellowships at Johns Hopkins. Dr. Oaklander works in the emerging field of nerve injury and neuropathic pain and is internationally recognized for her research, as profiled in Science and New Yorker magazines as well as in print and broadcast news media.
We are excited about Dr. Oaklander's research with the Foundation's grant and hope that her project will put us one step closer to improved diagnosis and treatment outcomes for Tarlov Cyst Disease. Dr. Oaklander’s publications should help educate the medical community and public alike about the diagnosis and treatment of Tarlov cysts.
Tarlov Cyst Disease Foundation Board of Directors
The Researcher Selection Process:
Requests for research proposals were sent out in March 2008.
Initial Research Proposals were received in May 2008.
NORD's Medical Advisory Committee extended invitations for final proposals from the research grant applicants July 2008.
Full Research proposals were received September 8, 2008.
Medical Advisory Committee selected the researcher/grantee November 2008!
Request for Proposals for Tarlov Cyst Research!
March 2008
The Tarlov Cyst Disease Foundation has provided a grant for restricted Tarlov cyst research. The term “restricted,” means that the grant is exclusively for researching Tarlov cysts. The Tarlov Cyst Disease Foundation is working with the National Organization for Rare Disorders (NORD) to determine the researcher, provide the grant and oversee the research process.
The process to determine the researcher/grantee began in March 2008 with NORD’s international request for proposals (RFP) and their existing database of researchers. The RFPs will be placed in medical and scientific journals and newsletters throughout the world. Individual mailings will be sent to academic researchers. Applicants may submit research proposals to NORD, no later than May 9 and apply for the grant the Foundation has provided. To read more about the research grant, visit the NORD website at : www.rarediseases.org/research/fp08/Tarlov_Cysts_2008_RFP.pdf .The NORD Medical Advisory Committee and the NORD Board of Directors will determine the final applicant, and the “grantee”/researcher will be announced on or around November 7, 2008. The grant is for a period of one year, and the researcher will submit the research results for publication to the scientific and medical community. The results of the research could be used to obtain further funding from NIH (National Institutes of Health) or other funding agencies. Providing this restricted Tarlov cyst research grant was a long-term dream that the Tarlov Cyst Disease Foundation members and donors helped to make possible. This is a significant initial step in the process to have a researcher focus on Tarlov cysts. The Foundation will continue seeking grants and more funding for research and education in the future; this is just the beginning!
If you wish to be part of the efforts provided by the Foundation, on behalf of all those around the world who live with the pain of symptomatic Tarlov cysts, contact the Foundation by writing to contactus@tarlovcystfoundation.org .You may also become a Foundation member or donor by visiting the “Join Us” page on this website.
We invite all Tarlov cyst patients to participate in the Patient Survey on the “Research & Survey” page of this website. These surveys will be provided to the grantee/researcher selected, so you will be part of the groundbreaking research that will provide solutions for the future!
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For the past two years, the Tarlov Cyst Disease Foundation has been working with the Honorable Congressman John J. Duncan, Jr.'s Tennessee and Washington offices to submit proposals to congress for appropriation of funding for Tarlov cyst research and education. The proposals were submitted in March 2007 and March 2008.
The Foundation had the assistance of the National Organization for Rare Disorders (NORD), the National Institutes of Health (NIH), The University of Tennessee Brain & Spine Institute and other individuals to create and support the proposal and the Foundation. The Labor, Health and Human Services, and Education Sub-Committee received, reviewed, and approved the initial proposal at the end of summer 2007. It was sent to the Appropriations Committee for their final approval, and if approved, for inclusion in the 2008 Congressional budget, after passing the full vote of Congress. The results of the 2009 Fiscal year proposal will not be announced until late 2009.
The Labor, Health and Human Services, and Education Sub-Committee membership represents 13 states with 17 members. Constituents, who have Tarlov cysts and who live in those states, contacted the committee members with the assistance of the Foundation. These Tarlov cyst patients are a part of the Foundation's Congressional Proposal committee, working closely with the Foundation's Board of Directors, to share important information about the proposal and Tarlov cyst disease, and to request their Congressional representatives' support of the proposal.
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Spring 2007
Tarlov Cyst Disease Foundation Hosts International Teleconference with Baxter International, Inc. and Physicians Who Treat Tarlov Cyst Patients
An incredible meeting of physicians to discuss treatments for patients with Tarlov cysts occurred in the spring of 2007.The Foundation arranged the meeting with Baxter International representatives from the Medical Affairs and Bio-Surgery Science divisions, in an unprecedented opportunity to discuss the use of the product Tisseel/Tissucol in surgical procedures, and in the procedure for aspiration and fibrin glue injection. The Tarlov Cyst Disease Foundation's President moderated the teleconference. Physicians who participated were from France, Italy and across the United States from the east coast to the west coast. This initial communication provided an opportunity for the physicians to share their treatment procedures for Tarlov cysts, and discuss possibilities for the future. There was a collegial harmony during the teleconference as they discussed their desire to share information with each other and to continue collaboration in the future. The teleconference was very beneficial as a communication between the company that manufactures Tisseel/Tissucol and the physicians who are using it to treat Tarlov cysts. The ultimate beneficiaries of this Teleconference are Tarlov cyst patients internationally.
Key summary points from the Teleconference:
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There was consensus among the neurosurgeons, interventional neuroradiologist, neurologist, orthopedic surgeon, and pain management specialist that changes in cerebrospinal fluid (CSF) pressure are a critical factor in the cause of Tarlov cysts and in the treatment.
It was discussed that a possible treatment consideration should be made, regarding a combination of aspiration and fibrin glue injection procedure and a shunt placement, to prevent future cyst development due to increase in CSF pressure. This was discussed due to new cyst development or refilling of cyst, that has occurred after surgery and aspiration and fibrin glue injection procedures.
The physicians were in agreement to share their data from patient treatments and outcomes in order to provide a scientific research analysis in the future. The Foundation will facilitate this project with a research company.
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Baxter International representatives made the following key points regarding the physiology of the product Tisseel/Tissucol:
* The FDA and Baxter recommends that Tisseel/Tissucol not be used more frequently than every 12 months in the same patient, due to the increasing possibility of an allergic reaction to the product. Tisseel has been used for 28 years in surgical procedures for heart, pancreas, liver and other organ surgeries as a tissue sealant.
* Tisseel is a biodegradable product that is physiologically designed to absorb in 7-14 days. Baxter International, Inc. has not tested the use of Tisseel in closed cavities such as inside a cyst, nor in a closed cavity containing cerebrospinal fluid.
* Tisseel, a tissue sealant product, physiologically reaches 70% of its clot forming potential in 3 minutes, and it reaches its full potential in 2 hours.
* There is nothing in the physiology of Tisseel that would be affected by the patient taking Non-Steroidal Anti-Inflammatory Drugs (NSAID), before or after the procedure using Tisseel.
Fostering communication and collaboration within the medical community is one of the Tarlov Cyst Disease Foundation's specific purposes and objectives. We believe the teleconference is a beginning of improved understanding between physicians willing to share their experiences. It is the Tarlov Cyst Disease Foundation's plan to provide a mechanism for future research endeavors with clinical investigators, industries that provide pharmaceutical products and devices, academic research centers, and others interested in scientific data collection related to Tarlov cyst patients and their treatments and outcomes.
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