FDA Advisory Committee Recommends New Treatment for FDA Approval
Finally there is a drug therapy pending approval by the FDA that specifically addresses improving walking function in people with MS. In mid-October, Fampridine, produced by Acorda Therapeutics, was recommended for approval by an advisory committee of the FDA. The FDA itself is expected to rule on the drug in January 2010.
In clinical trials, Fampridine helps with motor fatigue, and demonstrated an ability to improve walking speed by as much as 25 percent in people with any type of multiple sclerosis. The drug is a tablet form of 4 aminopyridine, which is a chemical legally dispensed by compounding pharmacies that was used in the past with some success. Fampridine is 4-aminopyridine formulated as a sustained release pharmaceutical, making it safer and more convenient to use. It improves the ability of nerve fibers stripped of myelin to conduct signals by blocking potassium channels on the nerve surface. The first research studies of this approach were funded by the National MS Society.
When myelin is stripped from a nerve, the potassium molecules inside the nerve fiber are exposed and attract calcium, which inhibits their ability to transmit signals, and can cause permanent damage.
While there are some side effects that can result from Fampridine, they appear to be mild and typically result in back pain, dizziness, insomnia, fatigue, nausea, balance disorder, and occasional anxiety. Fampridine is contraindicated in people with epilepsy.
The obvious good news is that while we all wait for science to unlock MS secrets in genetics and immunology, we are also developing more effective treatments in the present. Even a 25 percent increase in walking speed can translate into a tremendous improvement in quality of life for people with MS. Improved walking should mean improved ability to work and to maintain one’s independence, and with that comes greater peace of mind and overall well-being.
At the recent Stanley F. Waterman Research Lecture, held in conjunction with the Chapter’s Annual Meeting, Dr. Amit Bar-Or presented a significant new way for scientists to approach MS research.
Dr. Bar-Or delves deeply into research studies, beyond the ‘end-point’ used for FDA approvals, to understand the more complex functions of the immune system.
In some cases, disease modifying treatments might have unintended consequences that are negative, because they affect immune processes that we don’t fully understand.
For example, T cells have long been thought to be the primary culprit in demyelinization. But recent work done by Dr. Bar-Or and others indicates that B cells are also key players in the development of multiple sclerosis. In healthy people, B cells are rarely present in the cerebrospinal fluid while in MS patients, a clonally expanded B cell population is detected. B cells are immune cells that make antibodies and may play a role in the immune attack on brain and spinal cord tissues in multiple sclerosis. In fact, it may be a combination of T cells and B cells that are responsible for the nervous system damage in MS. Dr. Bar-Or’s studies into the underlying immune system processes at work in MS have changed the way the MS research community is thinking about multiple sclerosis and about opportunities for more effective treatments.
By John R. Richert, MD
There has never been a more exciting time in MS research than now. How are we planning to maintain critical discovery in such a challenging economic environment?
First, we are moving ahead. This year alone, we’re providing over $33.5 million to support 345 new and ongoing projects in our core research portfolio, plus $1.5 million for Fast Forward, our drug development subsidiary, which is also attracting its own new funding streams. Thanks to the efforts of our MS activists, an additional $5 million has been specifically set aside for MS research out of the 2009 Department of Defense budget.
Second, we have three clear research goals — Stop MS, Reverse the Damage, and End MS Forever. Here are some exciting projects making headway in our three-pronged approach:
- Our portfolio of projects focusing on immune aspects of MS, in particular the large-scale clinical trial of a pregnancy hormone, estriol, and Fast Forward’s recent investments in promising immune-modulating molecules are opening up new avenues to Stop MS.
- Our research on rehabilitation and health care delivery, and our four international teams working on nervous system protection and repair (funded by the Promise: 2010 campaign) are making tremendous progress, including plans for small-scale clinical trials to test the safety of cell therapy. These are examples of strategies to Reverse the Damage.
- The definitive scan for all common MS-related genes being performed by the International MS Genetics Consortium promises a wealth of data. This international collaboration launched by Society funding has the potential to help us find ways to End MS Forever.
And finally, we are prioritizing our spending to focus on what’s most important. Our choices—which we believe will be temporary—include:
- To continue to fund our discovery research portfolio and fellowships at reduced levels.
- To place some research award programs on hold.
We hope that the reduction will be of short duration. Should it become necessary to continue on this path, we know that some established researchers and some promising young scientists will abandon MS to move into areas where funding is more plentiful. We have to do everything possible to keep them in the MS field.
We are poised to make quantum leaps forward. Exciting new technologies, like gene chips, proteomics, and new imaging techniques are allowing scientists to visit age-old questions about MS in new ways. Now more than ever, we need the flexibility and resources to pursue the most promising leads.
Each of us plays a crucial role in fueling MS research and ensuring that the brightest minds continue to pursue the answers that we so anxiously await. Now is the time when each of us must take stock of our own priorities and ask, “What more can I do?” and “Who else do I know who could help?” Please act now!
- New Data Support Early Interferon Treatment for Multiple Sclerosis
In a study of 2,570 people with MS, early treatment with interferon therapy was associated with a significant reduction in the risk of MS progression.
- Fast Forward Think Tank Explores Personalized Medicine and the Translation of High-Tech Lab Discoveries to Improving Treatment of MS
As a result of this meeting, Fast Forward leadership plans to convene a small working group that will focus on next steps and identifying resources needed to create a shared framework for academic and industry groups to work together on issues related to biomarkers and genetics in MS.
- MS Trial Alert: Study of Oral Teriflunomide (HMR1726) Recruiting People with Relapsing Forms of MS Worldwide
The study is comparing two doses of teriflunomide, an investigational, oral MS medication, and inactive placebo. Teriflunomide is an agent that may decrease immune system activity in MS. Other studies of teriflunomide in MS are ongoing.
- MS Trial Alert: Recruiting Nationwide for Study of Copaxone in Patients with First Episode of Acute Optic Neuritis
The study is to determine whether nerve fiber loss can be minimized with administration of glatirmamer acetate treatment. Optic neuritis is an inflammation of the optic nerve, and often is the first symptom of multiple sclerosis.
- Harvard study links teen obesity in girls to increased risk of developing MS
A study of more than 200,000 female nurses suggests that those who were obese at the age of 18 were twice as likely to develop MS later in life. Women who had a BMI of more than 30 at age 18 were more than twice as likely to develop MS. Having a large body size (based on silhouettes) at age 5 or 10 was not associated with an increased MS risk, but a large body size at age 20 was associated with a 96% increase in MS risk. Adult BMI was not associated with increased MS risk. Based on this study in women, it is not clear whether the same would hold true for teenaged boys.
- Minimally Invasive Radiosurgery May Improve MS Facial Pain that Does Not Respond to Drugs
Researchers report that minimally invasive surgery using precision radiation (known as gamma knife radiosurgery) relieved facial pain known as trigeminal neuralgia in a group of 37 people who had not fully responded to standard treatments. This surgery does not involve incisions, but is performed by applying intersecting beams of radiation precisely focused on the area in the brain thought to be causing the pain. Its usefulness for treating MS trigeminal neuralgia has not been well studied.
- Society-Funded Researchers Show that Early Relapses Link to MS Progression
A group funded by the National MS Society reports that more relapses early in the course of MS were associated with increased disease progression in a study of 2,477 people with MS, but that this effect diminishes over time.
Clinical trials help determine whether a drug is safe and effective for MS. Without people living with MS who are willing to serve as volunteers in these studies, we could not look forward to new and better therapies. For more information, search the National MS Society’s Database of Clinical Trials Recruiting People with MS at www.nationalMSsociety.org/research/clinical-trials/participate-in-clinical-trials/index.aspx and explore these other sites and resources:
ClinicalTrials.gov A registry of clinical trials that are currently recruiting participants with many disorders in the United States and around the world. Searchable by disease.
CenterWatch www.centerwatch.com A publishing and information services company that provides information on clinical trials, including a list of MS studies currently recruiting patients.
NARCOMS www.mscare.org/cmsc/CMSC-NARCOMS-Information This registry of people willing to participate in MS research was initiated by the Consortium of MS Centers to facilitate multicenter studies.
MS-CORE www.ms-core.org The Multiple Sclerosis Cooperative Research Group (MS-CORE) provides information on collaborating investigators who foster multi-center, investigator-led clinical research aimed at improving treatments for people with MS. It was established with funding from the National MS Society.
MS International Federation www.msif.org/en/research/clinical_trials This site provides resources on all aspects of clinical trials related to MS, including participation and ethical issues.
CISCRP www.ciscrp.org The Center for Information and Study on Clinical Research Participation educates and empowers patients, medical and research professionals, the media, and policymakers about clinical research participation and what it means to be an active participant in the process. CISCRP also sponsors the Clinical Research Volunteer Community, an online community to share and learn from the experiences of those involved in clinical research.
ResearchMatch.org www.researchmatch.org A not-for-profit secure Web site, designed to provide people who are interested in participating in research the opportunity to be matched with studies that may be the right fit for them. ResearchMatch is a collaborative effort of the national network of medical research institutions affiliated with the Clinical and Translational Science Awards, a part of the National Institutes of Health.