June 2012 - The ALS Association Greater Sacramento

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Research Update Page 8 Continued from page 6. The ALS Association invited Steve Perrin, Ph.D., of the ALS Therapy Development Institute, to update the group on TDI's development of a therapeutic strategy targeting the body's immune system. Their work in mouse models has identified an immune pathway that appears to overactivate a certain type of T cell (one major type of immune cell), which carries a receptor called CD40. This activation leads to inflammation, which is thought to contribute to the disease process in ALS. TDI has worked to develop an antibody that blocks CD40, preventing activation of the T cell and increasing survival in mice. The antibody itself doesn't get into the central nervous system but may exert part of its beneficial effect by preventing other immune cells, called macrophages, from crossing into the central nervous system where they may do damage. Currently efforts are underway to develop a human antibody against the CD40 ligand. However, TDI demonstrated that the same pathway can be intervened by using an oral drug called fingolimod, which may have benefit in ALS patients. This drug, trade name Gilenya, was recently approved by the Food and Drug Administration for the treatment of multiple sclerosis. TDI is in early stages of planning a trial to test this compound in ALS. There are concerns about safety based on data from the use of the drug in multiple sclerosis. Dr. Perrin noted that the trial, which is still in the early planning stages, may require 24-hour monitoring of heart function to determine if treatment causes the heart irregularities seen in a small proportion of MS patients. Mark Boutin, Executive Vice-President and Chief Operating Officer of the National Health Council (NHC), discussed the MODDERN Cures Act (HR 3497), legislation endorsed by The ALS Association and introduced in Congress by Representative Leonard Lance (R- NJ). The bill would help accelerate the search for a treatment for ALS and other diseases by removing the barriers that limit medical innovation and by providing incentives to develop new treatments and diagnostic tools. He noted that despite increases in federal spending for medical research, the rate of new drug approvals has not increased. “Money is not the only barrier to development of new therapies,” Boutin said. As important are four barriers that inhibit the interest of pharmaceutical companies from taking risks in the development of new therapies. Addressing these barriers could rapidly change the development landscape. The first barrier, and the most important, is the problem of “intellectual property.” In the large majority of cases, it is difficult or impossible to obtain a patent on a potential therapy (this is true for all disorders, not just ALS). “We see this again and again,” said Boutin. Patents expire after a certain number of years, and the clock begins ticking not when the therapy is marketed, but at the beginning of a clinical trial development program. The development of a new drug, particularly for ALS, can take 15-20 years, reducing the potential return on investment; thus, many are not begun. The MODDERN Cures Act would create a new legal class of drugs called “dormant therapies,” which are those therapies that show promise in treating a disease with unmet medical needs, like ALS, but which have weak or no patent protection. Such dormant therapies would be afforded a period of “data exclusivity,” which offers incentives for development similar to those of patents. However, unlike patents, the period of data exclusivity would begin at the time of FDA approval so that manufacturers are not penalized for pursuing treatments for diseases like ALS that can take 15 or more years to develop. The other barriers relate to the ability to develop biomarkers and other diagnostic tools that can help speed treatment development by informing whether or not and in whom a drug may be effective much earlier in the development process. Even effective medicines work in an average of only 60% of patients because of genetic differences between individuals that affect response to the drug. “We have few effective tools to determine who a medicine will work for,” Boutin continued. Even when a diagnostic test is developed to determine differential response, it may take years to obtain the necessary medical coding from the government to allow reimbursement for the test. Nevertheless, the reimbursement is often too little to support the cost of the test. Regulatory and statutory changes included in MODDERN Cures could encourage development of such tests, speed the ability to obtain a reimbursement code, and better match reimbursement to costs. The National Health Council and The ALS Association are working with Congress, the FDA, the White House and industry to enact the legislation as soon as possible. “These are the kinds of efforts that we need to move our search for new treatments forward,” Dr. Bruijn said. “That is what all of us are focused on.”
How You Can Help Retire with Meaning - Help Your Kids & Fight ALS, Too How you Can Help? PAGE 9 Since you first started working, you’ve been regularly reminded of the importance of saving for retirement. Now you may find that your retirement savings are more than adequate. In addition, retirement assets are one of the most heavily taxed assets we own. Without proper planning, retirement assets may be subject to both estate and income taxes after you pass away. Donating all or part of a retirement plan to The ALS Association may be an attractive option. Since we are a charitable institution, we are exempt from paying the taxes that might otherwise be owed by your heirs and you will also receive an estate tax charitable deduction. This tax-wise move makes other assets you own available for loved ones. Making us the beneficiary of a retirement plan is so easy to do. Simply request a ‘Change of Beneficiary’ form from your advisor or the custodian of your account. Your plan administrator can send you a beneficiary designation form, which are often available online. The phone number and website of your plan administrator can found on your financial statement. Remember, you can name us as a partial beneficiary, the beneficiary of the entire amount or as a contingent beneficiary. Don't forget to incorporate the following legal description on the beneficiary designation: The Greater Sacramento (Tax ID 68-0159292), with its principal office in Sacramento, CA. A Very Special Thanks to the Following Sponsors for their Support of the 25th Anniversary Summer Soiree!
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Page 3 and 4: Walk to Defeat Update Page 3 The Wa
Page 5 and 6: Advice from the Trenches PAGE 6 San
Page 7: A Father’s Day Tribute I have bee
Page 11 and 12: Smooth Operators! VPD Rocking Beard
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June 2012 - The ALS Association Greater Sacramento

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