FDA Accepts New Drug Application for Potential ALS Treatment Edaravone

Following up on information released in June about edaravone, an intravenous drug therapy produced by Mitsubishi Tanabe Pharma Corporation, locally based in Jersey City, N.J., with a head office in Osaka, Japan, there has been recent news as to the status of the drug in the U.S.

Continue reading FDA Accepts New Drug Application for Potential ALS Treatment Edaravone

TREAT ALS Drug Discovery Contract Open Submission

Translational Research Advancing Therapies for ALS (TREAT ALS™) Drug Discovery Contract

Deadline for submission of Letter of Intent: Tuesday, July 10, 2012

PROGRAM RECEIPT DATES:
Letter of Intent
July 10, 2012
Request to submit full application
July 31, 2012
Submission of full application September 11, 2012
Award Announcements November, 2012
Funding commences on receipt of all relevant signatures

Email: researchgrants@alsa-national.org for forms or queries.

Program Description

There is currently one FDA-approved drug for the treatment of Amyotrophic Lateral Sclerosis (ALS), riluzole (Rilutek), improving survival by two to three months. Other treatments are available that relieve the symptoms associated with ALS and improve the quality of life for patients living with the disease by providing comfort to the patient. However, there is an urgent need for improved therapies. With the recent progress in understanding ALS, the increased effort to develop tools to identify novel treatments for the disease and advances in technology, the opportunity to discover improved treatments for ALS could not be better. The ALS Association’s Translational Research Advancing Therapies for ALS (TREAT ALS™) program funds research from early target identification to preclinical research and early pilot clinical trials. As part of the program, The Association is requesting letters of intent for its drug development contract program, milestone driven research to develop new treatments for ALS. The program complements the Department of Defense ALS research program and the translational programs at the National Institutes of Health.

Contract Information

The Drug Discovery for ALS Contract Program supports the preclinical assessment of therapeutics for ALS. The proposed studies are expected to be product-driven and focused on therapeutics. It is anticipated that the agents and/or data generated from these awards will lead to the advancement of new therapies for ALS. The program is designed to support preclinical testing and development of therapeutics for ALS. Applications must include preliminary data relevant to the phase(s) of the preclinical development process covered by the proposed research. The application should include a clear statistical plan of analysis, if appropriate. Applicants must clearly and explicitly articulate what impact the project may have on therapeutic development for ALS. Clinical trials will not be supported with this funding opportunity.
The contracts are limited to the areas of programmatic interest listed below. Applications must focus on one or more of these areas to be considered for funding. Applications that do not focus on at least one of the following areas will be administratively withdrawn. Preliminary data supporting the choice of target for drug development for ALS must be provided both in the letter of intent and the full application. Priority is given to applications focused on developing compounds directed towards the most attractive targets for ALS with significant data to support the relevance of the chosen target for ALS therapy.
  • Testing of compounds in mouse models of ALS.
  • Development of pharmacologic agents through Adsorption, Distribution, Metabolism, Excretion, and Toxicity (ADMET) phase;
  • Design and implementation of full-scale, pilot current Good Manufacturing Practice (cGMP) production of therapeutics and/or delivery systems for use in advanced preclinical and initial clinical trials;
  • Development of pharmacologic agents to the Investigational New Drug (IND) stage in order to initiate Phase I clinical trials after the award’s completion.
The preclinical drug development process may require resources beyond those available at a single organization. Therefore, the contracts are open to investigators participating in synergistic collaborations focused testing and developing lead agents for the treatment of ALS. Collaborations should be dedicated to a single, synergistic preclinical development project or study rather than an additive set of subprojects (i.e., the combined efforts of the collaboration must provide greater benefit than the sum of individual research initiatives). If a collaboration is proposed, letters confirming/supporting the collaboration are required. If the collaboration is multi-organizational, participating organizations will ensure the success of the collaboration by resolving potential intellectual and material property issues and by removing organizational barriers that might interfere with achieving high levels of cooperation. Biotechnology or pharmaceutical companies are encouraged to apply. Whether a biotechnology or pharmaceutical company applies for this mechanism as an individual applicant or as part of a collaboration, the company is expected to leverage its own resources to complement the funding provided by this contract.
Clinical trials will not be supported in this program.

Funding

  • The maximum period of performance is 2 years.
  • The maximum allowable direct cost for the entire period of performance is $500,000. The association does not provide indirect costs.
  • More cost-effective studies that do not request the full available funding amount are encouraged. The applicant may request the entire maximum funding amount for a project that may have a period of performance less than the maximum 2 years. (time-lines must be clearly described with go/no go milestones)
  • Regardless of the period of performance proposed, the applicant may not exceed the maximum allowable costs.
  • Travel costs of up to $1,500 per year to attend scientific/technical meetings are allowed
  • Salary for PI will not be supported.
Payment structure is based on achievement of milestones which will be finalized jointly with the investigators and the scientific advisory board if the study is approved for funding.

Letter of Intent
Due Date: July 10, 2012

Email researchgrants@alsa-national.org for form to be submitted.
Review Criteria for letter of intent.
1.    Scientific merit: Priority of therapeutic target as compared with other proposals
2.    Research Strategy and Objectives: How the scientific rationale supports the project objectives and feasibility.
3.    Impact: How the project will make an important contribution to ALS therapeutic development.
4.    Personnel: How the qualifications of the PI and key personnel are appropriate to perform the proposed research project.
5.    Consultants and Collaborators: How the overall collaboration will be most effective at achieving milestones and progressing the therapeutic towards clinical trials.
Notification of Pre-Application Screening Results July 31, 2012
Following the pre-application screening, PIs will be notified as to whether or not they are invited to submit an application; however, they will not receive feedback (e.g., a critique of strengths and weaknesses) on their pre-application.

Full Application due September 11, 2012

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The ALS Association and the Packard Center Partner to Develop Animal Model Systems for Most Common Cause of Familial ALS

The ALS Association and the Robert Packard Center for ALS Research at Johns Hopkins have entered into a partnership to expedite the development of animal model systems to expand the knowledge about the C9ORF72 gene, which has been identified as the most common cause of inherited amyotrophic lateral sclerosis (ALS or Lou Gehrig’s Disease) and Frontotemporal dementia (FTD).

“The Association is very pleased to partner with the Packard Center to expedite these important studies,” said ALS Association Chief Scientist Lucie Bruijn, Ph.D. “The Association along with the Packard Center have both invested significant funds into the identification of this new gene, and we are pleased to be able to work together to support the critical next steps to ensure that possible discoveries from these projects are translated as rapidly as possible into therapies for ALS.”

In October, 2011, a large expansion of a hexanucleotide GGGGCC repeat was discovered in the C9ORF72 gene, but how the expansion causes malfunction of the nerve cells in ALS and FTD remains unknown. It is thought that the messenger RNA (mRNA) derived from this large repeat aberrantly accumulates. This scenario is reminiscent of what is known in other diseases caused by expanded repeats, especially myotonic dystrophy.

Building on that example and in partnership with Isis Pharmaceuticals, the Cleveland Laboratory in San Diego, Calif., has designed a gene silencing approach to develop a drug called an antisense oligonucleotide (ASO) that will selectively destroy the ALS-causing mRNA with the expanded repeat.  Essential for drug development is a mouse model expressing the expanded human C9ORF72 mRNA.  The investigators will build these models and use them to validate efficacy of the ASO drug.  This research was funded by ALS Association California Chapters through a state program that allows taxpayers to direct donations toward the “ALS/Lou Gehrig’s Disease Fund” when completing state tax forms.

“Following the discovery of the most abundant genetic cause of ALS, we have initiated a drug development approach to selectively destroy the ALS-causing product of the mutated gene,” said Don W. Cleveland, Ph.D., Departmental Chair of Cellular and Molecular Medicine, University of California San Diego.  “The ALS Association partnership with the Packard Center will fund development of a mouse model that genetically mimics the human gene mutation, which will represent an essential tool for validating drug development.”

A parallel effort to inactivate the toxic C9orf 72 gene and identify drug activity biomarkers in ALS patients is also underway at the Packard Center with Bryan J. Traynor, M.D., of the Laboratory of Neurogenetics, National Institute on Aging and Johns Hopkins University and Jeff Rothstein, M.D., Ph.D., the Packard Center director.

“Our partnership with The ALS Association provides a fantastic opportunity to quickly follow up on the identification of C9ORF72 by Packard and ALS Association Investigators and rapidly provide the ALS community with the essential tools to study the disease and develop much needed therapies. We are pleased to join forces-once again-to collaborate on this critical project,” notes Packard Center Scientific Director Piera Pasinelli, Ph.D.

In addition, the mouse model may develop an ALS-like disease, which can be used to determine exactly what goes wrong in the presence of the aberrant mRNA. Complementary mouse models will be generated by a group of researchers from Johns Hopkins, which will focus on developing tools to understand the mechanisms resulting from the abnormally expanded repeats on C9ORF72 and point to new directions for the treatment of this devastating disease.

“I believe that this unique joint funding from The ALS Association and Packard Center will be critical for initiating studies to clarify how hexanucleotide repeat expansion in C9ORF72 causes neurodegeneration in ALS-FTD, and these efforts will impact the design of appropriate therapy for patients,” said lead researcher Philip C. Wong, Ph.D., Johns Hopkins School of Medicine Departments of Pathology and Neuroscience

Initially, Dr. Wong and his colleagues plan to develop mouse model systems to find out if losing C9ORF72 gene activity is responsible for death of motor neurons. Secondly, they hope to learn whether toxicity arises through the presence of a toxic RNA derived from the diseased C9ORF72 gene that determines malfunction of motor nerve cells.

Outcomes from these studies will clarify how hexanucleotide repeat expansion in the C9ORF72 gene causes motor nerve cell loss in a large proportion of cases of ALS. In addition, these efforts will have important implications for therapy design and provide useful mouse model systems for testing therapies that could eventually benefit people with ALS.

The mouse models developed through this initiative will be made rapidly available to researchers through the ALS Mouse Repository at Jackson Laboratories funded by a partnership with The ALS Association, Tow Foundation and ALS Therapy Alliance. Click here for more information.

The discovery of the C9ORF72 genetic repeat, made by Dr. Bryan Traynor was funded by The ALS Association and the Packard Center. Another study reported at the same time with similar findings was led by Rosa Rademakers, Ph.D., Mayo Clinic; this study was also funded by The ALS Association.  For additional information about the C9ORF72 studies click here and here.

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Olesoxime Phase III Trial Results Disappointing

Trophos SA has announced the results from the Phase III study of its lead compound, olesoxime, did not demonstrate a significant increase in survival versus placebo in 512 patients with ALS also receiving riluzole (Rilutek®).

The drug was well tolerated; however, it is believed that this outcome is most likely because in ALS the disease process is already so severe and rapidly progressing by the time of diagnosis that any further benefit of olesoxime over that of riluzole cannot be detected. This trend was measured by the ALSFRS-R functional rating scale.

“Although these results are disappointing, the ALS investigator community continues to test a variety of different approaches to treat ALS with several trials ongoing,” said ALS Association Chief Scientist Lucie Bruijn, Ph.D. “The Association encourages people with ALS to continue to enroll in ALS trials with a hope that one or more will provide important discoveries and lead to effective treatments for the disease.”

For more information about this announcement, click here.

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Ceftriaxone Study Information

Study Rationale

Ceftriaxone is a semisynthetic, third generation cephalosporin. Cephalosporins are beta-lactam antibiotics approved by the FDA for the treatment of infections such as septicemia, pneumonia, meningitis, and urinary tract infections. Ceftriaxone has been demonstrated to upregulate the expression of glutamate transporters in brain and has antioxidant properties. Ceftriaxone prolongs survival of a transgenic mouse model of ALS [1]. Preclinical evidence suggests that the extracellular accumulation of excitatory amino acids (EAAs) such as glutamate and the excessive activation of EAA receptors contribute to the neuronal cell death observed in both acute insults to the CNS and chronic neurodegenerative diseases. EAA transporters maintaining normal glutamate concentrations in the synaptic cleft typically prevent this excitotoxic result. Decreased expression of glutamate transporters EATT2/GLT-1 in patients with ALS and in mutant transgenic SOD-1 models has been observed. Conversely, animal models have shown that overexpression of these glutamate transporters can delay onset and prolong survival in ALS mice, and prevent motor neuron degeneration in vitro.

Study Goal

The study objective is to determine the pharmacokinetics, safety, and efficacy of long-term ceftriaxone treatment in subjects with ALS.

Study Status

The study is currently recruiting patients at many locations in the US and Canada, including at Beth Israel Medical Center, New York, New York. The Investigator for the study at Beth Israel Medical Center is Dr. Stephen N Scelsa.

Contacts

For more information, please contact Theresa Imperato, Study Coordinator, at 212 or imperator@als-ny.org for more information regarding this study.

REFRENCES

1. Rothstein, J.D., S. Patel, M.R. Regan, C. Haenggeli, Y.H. Huang, D.E. Bergles, L. Jin, M. Dykes Hoberg, S. Vidensky, D.S. Chung, S.V. Toan, L.I. Bruijn, Z.Z. Su, P. Gupta, and P.B. Fisher, Beta-lactam antibiotics offer neuroprotection by increasing glutamate transporter expression. Nature, 2005. 433(7021): p. 73-7.

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AAN Guideline Evaluates Treatments for Muscle Cramps

From The American Academy of Neurology Website.

ST. PAUL, Minn. – A new guideline from the American Academy of Neurology recommends that the drug quinine, although effective, should be avoided for treatment of routine muscle cramps due to uncommon but serious side effects. The guideline is published in the February 23, 2010, issue of Neurology®, the medical journal of the American Academy of Neurology.

“It’s important for people to know that quinine should be avoided since the drug is still available in some countries,” said lead guideline author Hans D. Katzberg, MD, of Stanford University and a member of the American Academy of Neurology. “Quinine should be considered only when cramps are very disabling, when no other drugs relieve the symptoms, and when side effects are carefully monitored. It should also be used only after the affected person is informed about the potentially serious side effects.”

The guideline found that naftidrofuryl, diltiazem and vitamin B complex may be considered for use in the treatment of muscle cramps, but more research is needed on their safety and effectiveness.

Read more.

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FDA Lifts Ban on Development of Arimoclomol

The Food and Drug Administration (FDA) has lifted a nearly two-year suspension on development of arimoclomol as a treatment for ALS, or Lou Gehrig’s disease, according to a report in Associated Press and the company developing the drug, CytRx Corp.

The FDA previously approved a revised clinical trial design for the drug candidate.

In January 2008, the FDA halted arimoclomol studies, citing the need for additional analysis from previously completed animal studies with arimoclomol. In June 2008, CytRx said it would have to conduct more animal toxicology studies on arimoclomol.

Results from the new trial could be available 18 months after the study starts, according to CytRx.

A little information about arimoclomol from the national ALS Association website:

Arimcolomol acts by upregulating heat shock proteins. It is effective in the SOD1 mouse model of ALS even when initiated after the onset of symptoms, and was found to be safe and well tolerated in a previous phase II clinical trial that included patients with sporadic ALS.

Previously, medications that have been found to be effective in the mouse model of ALS have not shown benefit when brought to human clinical trials. Investigators at Emory University in Atlanta and the Massachusetts General Hospital (MGH) in Boston, led by Drs. Michael Benatar and Merit Cudkowicz, believe that the SOD1 mouse model of ALS most closely resembles SOD1-positive familial ALS in human and hence this is the population most likely to benefit from arimcolomol.

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2010 Medicare Rx Drug Benefit Open Enrollment – November 15 to December 31, 2009

Click here to open the full Medicare Drug Benefit Guide in a printable version. Note: This requires Adobe Acrobat Reader.

The annual open enrollment period for the Medicare prescription drug benefit is underway and will continue from November 15 until December 31, 2009. During this period, those currently enrolled in a Medicare prescription drug plan have an opportunity to switch plans, or they can remain in their current plan. Those who did not enroll in the benefit when they first became eligible for Medicare also may enroll at this time, although these individuals may be subject to a late enrollment penalty.

It is important that PALS who have enrolled in the Medicare drug benefit take the time to review their prescription drug plan options, even if they are satisfied with their current plan. Many plans have made important changes to their benefits for the upcoming year, including changes to monthly premiums, the drugs that are covered or included on the plan formulary, the costs of drugs, coverage in the “donut hole” and other policies that impact access to particular drugs.

In addition, new plans with different options are now available in many areas of the country. Therefore, your current plan may or may not be the best plan for you, so we encourage you to take the time to review your options and find the plan in your area that best meets your needs. And as you review your plan options, we strongly recommend that you evaluate plans taking into account a range of factors, such as coverage policies and your drug needs in addition to monthly premiums.

As we have done every year since the drug benefit was implemented, the Advocacy Department is providing helpful information, including tips and tools, which you can use in selecting a Medicare prescription drug plan. This information includes.

* Riluzole Coverage
* Costs, Coverage & Choices
* Choosing a Prescription Drug Plan
* Extra Help is Available
* 14 Questions and Answers about Open Enrollment
* Information to Have and Questions to Ask
* Report Problems and Successes

Importantly, ALS Association Chapters as well as caregivers and families also can use this information to assist PALS in choosing and enrolling in a Medicare prescription drug plan. These resources will allow people to compare their current plan with other plans and to identify a plan that covers specific drugs, like Riluzole, that are needed by people with ALS. They also include tips on questions to ask when selecting a plan, as well as answers to many of the questions you may have about this year’s open enrollment.

If you have any questions about this information or the Medicare prescription drug benefit, please contact patient services at (800) 672-8857 or email at patient_services@als-ny.org.

Medicare Prescription Drug Information & Resources

Open Enrollment:
Nov. 15 through Dec. 31, 2009

Riluzole Coverage

People with ALS do have coverage for Riluzole through the Medicare prescription drug benefit in 2010. Thanks to The ALS Association’s outreach, the drug has been covered by Medicare plans since the prescription drug benefit was first added to Medicare. However, coverage may vary, including copays and network pharmacies, so it is important to evaluate these factors when deciding on a plan.

It is important to note that Medicare prescription drug plans may not discriminate against beneficiaries on the basis of their medical condition. Because Riluzole is the only drug approved to treat ALS, plans effectively would discriminate against PALS if they denied coverage for the medication. Therefore, if you are unable to identify a plan in your area that covers Riluzole, please contact the Advocacy Department (advocacy@alsa-national.org or 1-877-444-ALSA) so that we can immediately bring this to the attention of the Centers for Medicare and Medicaid Services (CMS) and ensure that you will have access to Riluzole. Please also let us know if your Rx drug plan imposes additional restrictions on coverage for Riluzole, such as higher copays or prior authorization requirements. This feedback is important and will help us to resolve any problems PALS experience accessing the only drug approved for the treatment of ALS.

Costs, Coverage & Choices

Costs and coverage will vary depending on where you live and which plan you select. For example, plans will have different monthly premiums, annual deductibles and copays. There also will be differences in which drugs are covered, the cost of specific drugs, policies related to prior authorization, and choice of pharmacy among other policies. In addition, some plans will pay drug costs during the so-called “coverage gap,” the portion of the standard Medicare drug benefit where you are responsible for paying all costs for your drugs before becoming eligible for catastrophic coverage. Because of the differences in each plan available to you, it is vital that you evaluate your choices to determine which plan best meets your needs.

If you already have Medicare coverage and are satisfied with that coverage, you do not need to do anything in order to maintain coverage. However, we urge you to review your options to make sure you are aware of any changes your current plan may be making in 2010.

Choosing a Prescription Drug Plan

There are several resources, both online and over the telephone, available to assist you in choosing a Medicare prescription drug plan and to compare your plan with others available in your area. They include:

Medicare Website Tools: Available on the Medicare website, www.medicare.gov, are several important on-line tools, including:

* Medicare Prescription Drug Plan Finder. The Plan Finder will allow you to identify plans in your area and to compare your plan with others available to you. Importantly, the Plan Finder will identify which plans cover the drugs you take and includes features that provide you with an estimate of your out of pocket costs for each plan you review, including the costs of specific drugs. Information on estimated monthly mail order drug costs compared to retail drug costs also is available. The Plan Finder also includes ratings of plans based on a number of different factors.
* Formulary Finder: The Formulary Finder enables you to identify plans in your state that cover the drugs you need.
* Lower Your Costs During the Coverage Gap: This section of the site provides tips on how you can lower your out-of-pocket costs, including through assistance program

MyMedicareMatters.org
Another helpful tool that is available to PALS, Chapters, caregivers and family members is www.MyMedicareMatters.org. This web-based decision-making tool is provided by the National Council on Aging and the Access to Benefits Coalition. New to the website this year is a section called, “Review 2010 Choices,” that was created especially for those who have a current Medicare prescription drug plan. This section focuses on why someone should consider changing plans, when it is appropriate to do so and how to pick a new plan. Those who are looking to join a plan for the first time can “Start with 7 Simple Steps,” another feature on the site. This section explains eligibility, how to evaluate current drug coverage, cost, and how to pick a plan. Both sections link to the www.Medicare.gov Prescription Drug Plan Finder and there’s also a downloadable instruction page that outlines how to use the Plan Finder tool. (http://www.mymedicarematters.org/PrescriptionDrugs/Instructions/instructions.asp). Finally, the website includes information on how you may be able to find extra
help paying for your prescription drugs.

Medicare Hotline, 1-800-Medicare
Medicare’s toll-free hotline is available to provide answers to questions as well as personalized assistance identifying and reviewing your prescription drug plan options.

Medicare & You 2010
Information about the Medicare program, including the prescription drug benefit, can be found in the 2010 edition of the Medicare & You handbook, which was mailed to all Medicare beneficiaries in October, 2009. The handbook also is available online at www.medicare.gov. The handbook includes tips on selecting a plan and an overview of plan options. Those who already are enrolled in a Medicare drug plan also should have received an Annual Notice of Change that describes any changes in the benefits offered by your current plan.

State Health Insurance Assistance Program (SHIP)
Each State has a State Health Insurance Assistance Program that offers free one-on-one counseling to PALS and other Medicare beneficiaries and their families. To find the SHIP office near you, go to www.shiptalk.org.

MAPRx
Medicare Access for Patients Rx, MAPRx, is a coalition of patient, family caregiver and health professional organizations committed to safeguarding the well-being of patients with chronic diseases and disabilities under the Medicare prescription drug benefit. The ALS Association has worked with the coalition; and the MAPRx website, www.maprx.info, includes helpful information and answers to questions. It also includes a list of resources to turn to in your state for assistance.

MyMedicareCommunity.org
Another helpful site available from the National Council on Aging and the Access to Benefits Coalition is www.mymedicarecommunity.org. The site is designed for professionals and volunteers who work with people with Medicare. Resources include articles, news items, calendars and easy-to-access Medicare references, including regulations and policy guidance from such sources as the Centers for Medicare & Medicaid Services (CMS), the Social Security Administration (SSA) and the Administration on Aging (AoA). A separate “forum” gives users the opportunity to ask questions, share ideas and promising practices, and try to help each other solve problems.

Extra Help is Available

Extra Help in paying for Medicare prescription drug costs is available for those with limited incomes, including through the Social Security Administration and other public and private sources.

Social Security
Under the Medicare Prescription Drug benefit, you may be eligible for the Extra Help program to pay for all or most of the premiums, annual deductible and copayments. You automatically qualify for Extra Help if you receive Supplemental Security Income (SSI) and have Medicare; receive Medicaid and have Medicare; OR your state pays your Medicare premiums. You also may qualify and can apply for Extra Help through the Social Security Administration (SSA), www.ssa.gov. In general, a person with Medicare may qualify for Extra Help if:

* Annual income is less than $16,245 for a single person or less than $21,855 for a married couple living together. Slightly higher income limits apply if you receive financial support from other relatives living in your household, if you receive earnings from work, or if you reside in Hawaii or Alaska.
o Social Security does not count the following as income: food stamp assistance; home energy assistance; medical treatment and drugs; housing assistance; disaster assistance; earned income tax credit payments; victim’s compensation; scholarships and education assistance
* In order to qualify for Extra Help, your total resources or assets, such as savings accounts or investments, must be limited to $12,510 for a single person or $25,010 for a married couple living together. SSA does not count a person’s house or car as resources.

Even if your income or assets are slightly higher than the limits listed above, you should still apply for Extra Help. The Social Security Administration allows other deductions that may make you eligible for Extra Help. and select the section on Medicare at the top of the page to see if you are eligible for Extra Help. Additional information also is available here: http://www.ssa.gov/pubs/10115.html.

BenefitsCheckup.org
This online tool (www.benefitscheckup.org) is a service of the National Council on Aging and will allow you to search for private and public programs that can help you pay for prescription drugs and other health care costs.

Pharmaceutical Assistance Programs
Many pharmaceutical manufacturers offer assistance programs for people enrolled in the Medicare Drug benefit (and for those who are not enrolled).

* Partnership for Prescription Assistance. Another coalition of pharmaceutical companies, providers and charitable organizations, the Partnership for Prescription Assistance (www.PPARx.org) offers a single point of access to more than 475 public and private patient assistance programs, including programs that offer assistance with Rilutek.
* Medicare Pharmaceutical Assistance Program Site: visit http://www.medicare.gov/pap/index.asp, to search manufacturer assistance programs by drug name.
* TogetherRxAccess.com: This coalition of drug makers provides assistance for drugs used by people with ALS. Visit www.TogetherRxAccess.com.
* State Pharmaceutical Assistance Programs: At least 23 states offer assistance with paying drug plan premiums and/or other drug costs. To learn if your state has a program, visit http://www.medicare.gov/spap.asp.

NOTE: Assistance programs have eligibility requirements (eg, in addition to income/resource limits, some are not available to those who are eligible for Medicare)

Frequently Asked Questions and Answers for the 2010 Medicare Drug Open Enrollment and Four Questions You May Have After Enrollment

The ALS Association once again has joined as member of MAPx (Medicare Access for Patients Rx) to provide answers to several important questions you may have about the Medicare Drug benefit as well as questions you may have after enrolling in a Medicare drug plan. A printable brochure can be found here:

http://www.maprx.info/pdf/9195_MAPrx_Open_2009.pdf

1. Will my Medicare prescription drug plan be the same in 2010 as it was in 2009?
Probably not. Almost all Medicare Part D plans will change in 20109. Use this open enrollment time to compare plans and find the plan that best meets your prescription drug needs at a cost you can afford.

2. How may my plan change in 2010?

Your current plan may have changed:

* monthly premium;
* annual deductible
* your share of the costs (copayment or coinsurance);
* the list of drugs it covers (formulary);
* coverage, if any, it offers in the coverage gap; and/or
* use of policies that may restrict access to certain drugs, such as:
o requires your doctor to justify why you need a certain drug before the plan will pay for it (called prior authorization);
o requires your doctor to prescribe a cheaper drug in the same class of drugs first (called step therapy); and/or
o only lets you buy a certain amount of a drug at a time (called quantity limits).

Your plan may also decide not to participate in 2010. If that is case,
your plan sent you a letter in early October explaining that you will need to select a new plan. You can pick a new plan between October 2009 and January 2010 as part of a Special Enrollment Period.

3. How do I know what changes my plan is making for 2010?

You should have received a letter from your current plan called an “Annual Notice of Change” by October 31. This letter explains some of the important changes to your plan, including changes to the premium, the drugs covered (formulary), the cost of the drugs, and any restrictions used that limit the access to drugs. If you did not receive the Annual Notice of Change letter, call your plan immediately.

While very important, this letter probably does not have all the details you need to determine if your current plan is the best plan for you in 2010. You also need to know how these changes apply to the drugs you use. You can find this information by looking on the plan’s Web site or in the Medicare Prescription Drug Plan Finder at www.medicare.gov or by calling the plan or 1-800-MEDICARE; (1-800-633-4227 / TTY: 1-877-486-2048).

You may have received a summary of the formulary with the Annual Notice of Change letter. If you did not receive a copy of the formulary, call the plan and they will send you a copy or tell you if your drugs are covered. The phone number for the plan’s customer service department is included in the Annual Notice of Change letter you received. You may also get information about the formulary from the plan’s website, by using the Medicare Prescription Drug Plan Finder at www.medicare.gov, or by calling 1-800-MEDICARE (TTY: 1-877-486-2048).

4. Should I compare my plan with others available in my area in 2009?

Yes, this is very important to do. Other plans may provide you with better or less costly coverage for the drugs you need. Often the single most important factor in choosing a plan is comparing the drugs you take to the plan’s formulary. The lack of coverage for one drug for a chronic condition can be the most important factor in terms of what your drug costs will be. The best way to compare your current plan with other plans is to use the Medicare Prescription Drug Plan Finder at www.medicare.gov -in the Prescription Drug Plans box, click on “Compare.” The Plan Finder will allow you to see the estimated costs for your current plan in 2010 and to compare those costs with other plans in your area. Estimates are based on drug prices on the date you compare plans; your actual out-of-pocket costs may varyDrug Plans box, click on “Compare.” The Plan Finder will allow you to see the estimated costs for your current plan in 2009 and to compare those costs with other plans in your area. Estimates are based on drug prices on the date you compare plans; your actual out-of-pocket costs may vary.

An important feature on the Plan Finder is an estimate of your total monthly costs over a 12-month period for each of the plans that you are considering. If you have entered the drugs you take, this information appears in a chart near the bottom of each plan’s Plan Drug Details page in a section titled Total Monthly Cost Estimator. Click on the name of your current plan to pull up the Plan Drug Details page.

5. What does it mean if a plan offers “coverage in the gap”?

The coverage gap is also called the “donut hole.” The coverage gap is a period during which you have to pay all the costs for your drugs AND continue to pay your monthly premium to keep your coverage.

  • The coverage gap begins after you and the plan together have spent a certain amount (no more than $2,830) on drugs that are included in the plan’s formulary and bought at a pharmacy in the plan’s network.
  • The coverage gap ends after you and your plan together have spent $6,440 in total drug costs paid. This amount includes the $3,610 that you spend on drug costs during the coverage gap. Only money spent on drugs on the plan’s formulary that are bought at a pharmacy in the plan’s network counts toward the coverage gap totals. After $6,440 has been spent, you qualify for catastrophic coverage—at which time you will pay only your monthly premium and up to 5% of your drug costs. [See http://www.maprx.info/pdf/MAPRx_Open_Enrollment_QAs_2008_Post.pdf.]
  • None of these amounts include what you spend on your monthly premiums.

Some plans provide coverage in the coverage gap, but most do not. In 2010, this means that such a plan will continue to provide some coverage for generic drugs during the coverage gap. Plans with coverage in the gap for generics are available in every state. Before enrolling in a plan with coverage during the gap, it is important to check with the plan to make certain the drugs you need are covered during the gap. The Plan Drug Details page on the Plan Finder will show your estimated monthly costs for each plan you are considering, how those costs will or will not change during the coverage gap, and by how much. Plans with coverage in the gap may charge a higher monthly premium.

Depending on your prescription drug needs, plans with coverage in the coverage gap may not save you money and may end up costing you more due to higher premiums and cost sharing.

If you get Extra Help (low-income subsidy) paying your drug costs, you won’t have a coverage gap. However, you will have to pay a small co-payment or coinsurance amount for each prescription until you reach catastrophic coverage.

6. What happens if a drug I take is not on a plan’s formulary?

You must pay the full cost for any drug not on the formulary. The money you pay for these drugs does not count toward the total amount that you must spend to qualify for catastrophic coverage. That is why it is important to make sure that your drugs, especially the most expensive ones, are on the formulary of the plan you select. You, your authorized representative or your doctor can ask for a “coverage determination” (exception) to get your plan to cover a drug when it is not on the plan’s formulary. See question #3 in the next section of this document for more details.

7. What do I have to do if I decide that I want to stay in my current plan for 2010?

Nothing. You will stay enrolled in your current plan unless you sign up for a new plan.

8. If I decide to change plans, how and when should I do it?

You can enroll in a new plan by contacting the plan you want to enroll in or by calling 1-800-MEDICARE (1-800-633-4227 / TTY: 1-877-486-2048) or by visiting www.medicare.gov.

You can change your plan for 2010 by enrolling in a new plan between November 15 and December 31, 2009. However, it is best to make the change as early as possible to ensure that you can get the prescriptions you need without delay on January 1, 2010. There is no fee for changing to a new plan. Do not disenroll from your 2009 plan if you enroll in a new plan for 2010. You will automatically be disenrolled from your 2009 plan when you enroll in a new plan for 2009. (On page 4 here, http://www.maprx.info/pdf/9195_MAPrx_Open_2009.pdf, is a chart on the Enrollment Period and Options)

9. If I’m in a Medicare Advantage Plan with drug coverage, but am not happy with the health coverage, can I drop my Medicare Advantage Plan and return to Original Medicare by itself and add a drug plan?

Yes, you can switch plans during the Part D Open Enrollment Period from November 15 through December 31, 2009.

You can also switch plans during the Medicare Advantage Open Enrollment Period from January 1 through March 31, 2010. During this period you can switch from your Medicare Advantage plan with drug coverage to the Original Medicare Plan but you must also join a separate stand-alone drug plan. The booklet Medicare & You 2010 has important information about Medigap protections for people switching from Medicare Advantage plans to Original Medicare.

10. What if I change plans, but find that I don’t like my new plan?

In general, you can only switch to another plan from November 15 to December 31 each year. However, there are a few special exceptions, such as if you move out of the service area, lose your employer drug coverage, enter or leave a nursing facility, or if you qualify for Extra Help. That is why it is so important to review your options before enrolling.

11. If I previously applied and qualified for Extra Help (Low-Income Subsidy), do I qualify in 2009?

If you applied and qualified for Extra Help at any time and are receiving Extra Help now, Social Security may have contacted you to review your eligibility status for 2010. In late August 2009, Social Security mailed letters to people who were selected for review and included a form to complete called “Social Security Administration Review of Your Eligibility for Extra Help” (Form SSA-1026).You had 30 days to complete and return this form. Any changes in the amount of Extra Help you will receive will be effective in January 2010.

If you qualified for Extra Help in 2009, but were not selected for a review, you will not receive a form from Social Security and there should be no change in the amount of Extra Help you receive. If you are unsure of your Extra Help status, call 1-800-MEDICARE (TTY: 1-877-486-2048).

If you have been notified by Social Security that you are no longer eligible for Extra Help in 2010, you will still be enrolled in your plan. After January 1, 2010, you will have to pay monthly premiums and your share of the drug costs. However, during a one-time Special Open Enrollment period, you can change Part D plans between January 1 and March 31, 2010. This will be an important opportunity for you to change to a new plan if you find that your existing plan is not your best option.

11b. If I automatically qualified for Extra Help in 2009, will I qualify in 2010?

If you automatically qualified for Extra Help in 2009 you will continue to automatically qualify in 2010 if you:

  • Receive both Medicare and Medicaid;
  • Have your Medicare Part B premiums paid by your state because you belong to a Medicare Savings Program; or
  • Receive both Medicare and Supplemental Security Income (SSI).

Medicare beneficiaries who automatically qualified in 2009, but who will not automatically qualify in 2010, should have received a notice on grey paper from Medicare [CMS Publication No. 11198] in September 2009.

The notice explains why you no longer automatically qualify and will encourage you to complete an enclosed Social Security application for Extra Help as soon as possible. The application for Extra Help should be returned to Social Security in the postage paid envelope provided.

12. Have the rules for Extra Help changed in 2010?

Yes. Social Security will no longer count life insurance you have as a resource when deciding if you qualify for Extra Help. They will also not count help you receive from others with your household expenses to decide if you get Extra Help.

You should know though that some states may still count life insurance and the help you receive from others to decide if you are eligible for your state’s Medicare Savings Programs (MSP). These programs can help pay for your Medicare Part B premiums and other Medicare costs.

If you applied for Extra Help in the past and were turned down because your income or savings were too high, these changes mean that you may be able to get Extra Help in 2010 if you apply again after January 1, 2010. Call 1-800-772-1213 or visit www.socialsecurity.gov or www.benefitscheckup.org.

If you apply for Extra Help, Social Security will send the information to your state’s Medicaid agency to start the process for getting you into your state’s MSP. If you do not want your information to go to the state, there is a box you can check on the application for Extra Help.

13. If I received Extra Help in 2009 and qualify again in 2010, will my drug costs change?

Probably yes. You will have an increase in drug co-payments of no more than 30 cents per prescription. In addition, the co-payment levels for some individuals will increase or decrease as a result of a change in their income or assets, or if they enter or leave a nursing facility or other institution.

If you continue to automatically qualify for Extra Help but your co-payment levels are changing in 2009, you should have received a letter on orange paper from Medicare [CMS Publication No. 11199] in early October telling you your new co-payment amounts.

14. What if I did not join a Medicare Part D plan when I was first eligible, but I would like to join one now?

You can enroll in a plan during Open Enrollment. You may have to pay a premium penalty if you did not have coverage that is at least as good as Medicare’s coverage (“creditable coverage”) during the first/initial period that you were eligible to enroll. The penalty amount is calculated based on the number months you were eligible but did not enroll. If you have to pay a premium penalty, most people will have to pay it for the rest of their life. The penalty will be added to your monthly Medicare private Part D plan premium.

If you qualify for Extra Help with your Medicare prescription drug coverage, you can enroll anytime and pay no late enrollment penalty.

15. Can I get free help to make decisions about Medicare prescription drug plans?

Yes. Every state has a State Health Insurance Assistance Program (SHIP) that offers free one-on-one counseling and assistance to people with Medicare and their families. SHIP offices are located throughout each state. To find contact information for the SHIP offices closest to your community, visit www.shiptalk.org or call 1-800-MEDICARE (1-800-633-4227 / TTY: 1-877-486-2048) for assistance.

Questions You May Have After Enrollment

1. I enrolled in a Part D plan but I haven’t heard anything. Is this normal?

No. You should have received a welcome letter and a prescription card from the plan. Contact the plan right away to confirm that you are enrolled.

2. I enrolled in a drug plan in December and got a letter welcoming me into the plan, but nothing else. I have nothing to show the pharmacist. How can I get prescriptions filled without a card?

Contact your plan immediately. If you need to get your prescription filled before your card arrives, bring the letter you received from the plan that confirms you have enrolled with you to the pharmacy. If you don’t have a letter, ask your pharmacist to call 1-800-MEDICARE (TTY: 1-877-486-2048).The customer service representative should be able to tell the pharmacist which plan you are enrolled in. If you continue to have problems, you should contact your local SHIP office. You can locate your local SHIP office by visiting www.shiptalk.org or by calling 1-800-MEDICARE (TTY: 1-877-486-2048).

3. Will my plan cover a drug that I need to take even if it is not on their formulary?

Maybe. You, your authorized representative or your doctor can ask for a “coverage determination” (exception) to get your plan to cover a drug when it is not on the plan’s formulary. Your plan can tell you how to do this. Your doctor can help you with some steps in the process. The plan must decide within 72 hours (or 24 hours for an expedited review) if they will cover the drug. If they decide not to cover the drug, they must send you a written notice. You also have a right to appeal their decision.

Note: If your drug is not on the formulary, but you are able to get it covered by the plan under the plan’s exceptions process, the money you spend on the drug is counted toward qualifying for catastrophic coverage. [See question #5 above.]

4. I am having problems with my old Part D plan. I have enrolled in a new Part D plan but my old plan still deducts a premium and it has been doing this for months now. I have called the old plan several times but I still can’t resolve the problem. What should I do?

Report billing errors to 1-800-MEDICARE (TTY: 1-877-486-2048) as well as to the plan. Since your plan has not stopped billing you after you notified it of the error, you may wish to file a complaint (grievance). Ask the plan’s customer service representative to send you a complaint form or tell you how to find one on the plan’s website. You can also file a complaint (grievance) with Medicare by calling 1-800-MEDICARE.

Information to Have and Questions to Ask

Regardless of whether people use the internet or more traditional means to identify a prescription drug plan, they should approach the process armed with important information and key questions to ask. CMS has available a helpful document that provide suggestions on issues to think about when comparing plans (http://www.medicare.gov/Publications/Pubs/pdf/11163.pdf). In addition, we have included below other information to consider when people review their options. But please remember that people should compare their current coverage to the new options that are available.

ALS Association Chapters may want to customize the information below to include any other questions PALS may want to consider as they go through the decision-making process.

General Information to Have On Hand When Choosing a Medicare Drug Plan:

1. Your Medicare Card, including Medicare number,
2. General information on your current prescription drug coverage,
3. Information on annual income and resources (to determine if you qualify for extra help).
4. A list of medications you take, including dosage.
5. The name of pharmacies you use.

Information to Consider:

The amount of the monthly premium

Whether the plan formulary includes

The particular drugs needed

The strengths and dosages of the drugs needed

The number of days covered in each prescription (Example: 30, 60, 90 days)

Whether
the pharmacies in the plan’s network include:

The pharmacies used by the beneficiary

The pharmacy used by the long-term care facility in which the beneficiary resides

Whether there are price differentials among pharmacies in the network
Whether mail-order is allowed or required
The price differential for mail order
The number of days covered in each prescription (Example: 30, 60, 90 days)
The plan’s utilization management tools
The prior authorization requirements
Whether the plan requires step therapy (Requirement that certain medication(s) be tried before those prescribed by the patient’s physician)
Whether the plan uses tiered cost sharing (Different co-pays for generics, brands, or for specific drugs)
The number of tiers
The co-payments/co-insurance per tier
Whether the plan offers therapeutic substitutions
Whether there are quantity limitations
On number of prescriptions in a month
On number of pills in a prescription
Whether the plan offers supplemental benefits
How the plan coordinates with the State Pharmaceutical Assistance Program
Who is the plan sponsor, has the entity been in the community for a while, is it reliable?
The “Transition” process used by the Prescription Drug Plan (Temporary use of a drug not covered by plan)
The “Exceptions” process used by the Prescription Drug Plan (Appeal if a person’s drug is not covered by the plan)
Whether a PALS has other insurance that covers prescription drugs:
Through a Medicare HMO or other Medicare Advantage plan. If so, the person must keep getting drug coverage through that plan if he wants to stay in that plan.
Through a retiree health plan. If so, has the former employer told the person whether the insurance is as good as or better than Medicare’s coverage (i.e., “creditable coverage”)? If it is creditable coverage, the person may stay in that plan without getting a late penalty on the premium if he later decides to change to a Medicare drug plan.
Through a Medigap (Medicare supplemental) policy? If so, has the insurer told the person whether the insurance is creditable coverage? If it is not, the person will have to pay a late penalty on the premium if he keeps his Medigap drug coverage and later switches to a Medicare prescription drug plan.
Individuals with coverage through the Veteran’s Administration, TRICARE, Federal Employee Health Benefit Plan, Railroad Retirement Board, Program of All-Inclusive Care for the Elderly (PACE), or Indian Health Service, may continue receiving prescription drug coverage through one of those plans if that coverage is as good as what is offered from Medicare prescription drug coverage.

Report Problems….And Successes

It is extremely important that you contact the Advocacy Department (advocacy@alsa-national.org or 1-877-444-ALSA) if you experience any problems or difficulties with your Medicare drug coverage, either during the enrollment process or with the coverage itself, after you enroll. For example, please let us know if you do not have a choice of plans that cover Riluzole or other drugs you and other PALS frequently need. Also let us know if your plan restricts access to these drugs, requiring you to first try another drug or pay a higher copay because the drug is not on the plan’s formulary or list of preferred drugs.

Your feedback is essential and will help us to communicate concerns to CMS and Congress and resolve problems that PALS may experience. We also want to know if you have had a positive experience with the drug benefit, including whether the benefit reduced your out-of-pocket drug costs or enabled you to access drugs for which you did not have prior coverage. Again, we want to communicate these successes to CMS and Congress so that they know what works and what types of policies are benefiting people with ALS.

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New “Antisense” Experimental Therapy Clinical Trial Announced

A new experimental therapy using an approach known as antisense, in which a drug is designed to shut down the RNA (Ribonucleic acid) that is responsible for the production of disease-causing proteins, is being prepared for a clinical trial in people with a familial form of ALS later this year. The clinical trial follows research funded by The ALS Association through TREAT ALS (Translational Research Advancing Therapy for ALS), our research pipeline that funds and facilitates the development of treatments for ALS based on important laboratory findings.

The research that resulted in the identification of this antisense drug was first funded by The ALS Association in 2003, and has been developed for the clinic through an academic/industry partnership. ALS Association-funded researchers Drs. Don Cleveland Richard Smith and Timothy Miller, in partnership with Isis Pharmaceuticals in Carlsbad, Calif., initiated experiments in a rat model of ALS to determine whether reducing the amount of SOD1 protein may be beneficial in treating the disease.

Initial research in rat ALS disease models demonstrated that the antisense drug inhibited the mutant SOD1 protein, resulting in prolonged life of the rats. Time of treatment for the rats was near onset of symptoms, reflecting the scenario for actual patients who often have definite and even advanced signs of motor neuron loss by the time of ALS diagnosis. Researchers hope that this therapeutic approach will provide a similar therapeutic benefit in people with familial ALS due to mutations in the SOD1 protein. The antisense approach could also prove valuable in treating other neurological disorders, such as Huntington’s disease.

Together with the biotech company Isis, led by Dr. Frank Bennett, Dr. Timothy Miller, Dr. Merit Cudkowicz and Dr. Richard Smith, the team has conducted the necessary research to submit an Investigational New Drug Application with the Food and Drug Administration (FDA) to test this novel approach in people with ALS. The application was recently submitted to the FDA. The ALS Association will provide funding for the clinical trial.

“This achievement, and the process of taking an idea from the laboratory to the clinic, underscores the importance of The ALS Association’s TREAT ALS pipeline and the financial support provided to the investigators,” commented Senior Vice President, Research and Development Lucie Bruijn, Ph.D. “The development of new treatments is an extremely challenging and costly process. It is only through the support of our generous donors that this type of research is made possible.”

The ALS Association’s research program brings together the best scientific minds from the research and biotech communities to focus on finding the cause of ALS, developing effective treatments, and ultimately, a cure. We are currently funding more than 80 studies around the world, partnering with the best minds in the scientific and biotech communities. To learn more about how you can support The ALS Association’s premier ALS research program and studies such as the antisense clinical trial, visit our website at www.als-ny.org.

People interested in learning more about the clinical study should contact the MGH Neurology Clinical Trial Unit at (877) 458-0631 or by email at mghneuroclinicaltrialsunit@partners.org.

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A Randomized Clinical Trial of Lithium Carbonate with Riluzole versus Placebo with Riluzole in ALS Shows No Benefit.

The ALS Association National News Update.

In February 2008, Dr. Francesco Fornai and colleagues at the University of Pisa, Italy, reported in a pilot study that lithium carbonate at dosages of 300-450 mg daily (titrated to a plasma level of 0.4-0.8 mEq/liter) combined with riluzole showed a large positive effect in people with ALS (Fornai, F., et al., Lithium delays progression of amyotrophic lateral sclerosis. PNAS, 2008.105(6): p. 2052-2057).

To further investigate lithium carbonate as a possible treatment for ALS, a randomized, blinded, multicenter trial of lithium carbonate with riluzole versus placebo with riluzole was conducted in people with ALS in the U.S. and Canada. The study used similar dosing to the Italian study. The study was conducted by the Northeast ALS (NEALS) and Canadian ALS (CALS) Consortia and was sponsored by the National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health, The ALS Association and the ALS Society of Canada. This unique collaboration between investigators and funding organizations resulted in a novel study design and expeditious execution of the trial to efficiently answer a critically important clinical question. Study leaders included Drs. Swati Aggarwal, Lorne Zinman, Jeremy Shefner and Merit Cudkowicz.

An interim analysis was conducted after enrollment of the 84th subject and presented to the NINDS Data and Safety Monitoring Board in September 2009. Based on the interim analysis the trial was stopped for futility. This study did not show the same beneficial effect of lithium carbonate on the progression of ALS as the prior pilot study conducted in Italy.

Although the results are disappointing, it was very important for the ALS community to quickly and efficiently determine if the large benefit first observed for lithium could be replicated in a well controlled trial. With the ongoing assistance and commitment of patient volunteers, researchers can now focus on other promising therapeutics for patients with ALS.

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