12 posts tagged “ALS research”

2018 National ALS Advocacy Conference: A recap from Team of Advisors members Rich & Paul

Posted 4 weeks ago by

Last month, Rich and Paul from this year’s Team of Advisors attended the 2018 National ALS Advocacy Conference in Washington D.C. to learn about the latest ALS research, network, and meet with members of congress on Capitol Hill. Just over 600 people attended the event, including 100+ pALS and 240 first-time participants.

The event started on Sunday with opening remarks followed by an afternoon session, reception, networking dinner and entertainment. Paul and Rich had the opportunity to share with pALS about PatientsLikeMe. Read on to learn how it went and what they accomplished.

(front row: Holly Crowder, Stacy Crowder, Kathy Tavano, Team of Advisors member Paul and Tanner Hockinsmith from the ALSA; back row: Stephen Reynolds from Senator Cronyn’s office)

Day 2: Research & Policy

  • Opportunities to participate in research: Rich (and other participants) gave saliva samples to the Centers for Disease Control (CDC) and blood to the National Institutes on Health (NIH).
  • Interview with Rich: Rich spoke about the ALS Registry Taskforce that exists to brainstorm and come together to find ways to increase participation.
  • Public Policy sessions: These sessions included time to prepare for the visit to Capitol Hill, a Congressional Panel with bipartisan Congressional staff and remarks from US Senator Bill Cassidy.
  • Interview with Pat Quinn: Pat Quinn, the Ice Bucket Challenge co-founder spoke about a new voice generating software and platform.
  • Research panel: Lucie Brujin, John Landers and Tody Ferguson held a panel discussion on where we are in ALS research, and what is coming in the pipeline.
  • Closing remarks/strategy: Following the closing remarks, attendees had time to strategize with state chapters. Paul and Rich’s chapter (the ALS Association FL Chapter) was represented by 17 people including:
  • 2 pALS, caregivers, family members, friends, the President of the ALS Association chapter in Florida and 3 researchers

 

(Tim Cummings (surviving spouse of ALS patient, former board president of ALS association FL chapter), Dorothy Marsh (who lost her sister to ALS), Martin Rivera (Rep. Darren Soto’s office), Team of Advisors member Rich, Talitha, and Rich’s daughter, Gigi.)

Day 3: To the Hill

Rich, Paul and the other members of their chapter headed to Capitol Hill to meet with senators, representatives and their staff.

Their mission?

The Taskforce of advocates asked U.S. Senators and Representatives to gain support for:

  1. The senate and house bills dealing with the ALS Disability Assurance Act. Passage of this bill would eliminate the 5-month waiting period for Medicare.
  2. Continued funding $10M for Department of Defense (DOD) ALS research
  3. CDC funding $10M for the National ALS Registry
  4. Continued NIH funding for $39.3B
  5. Support for Steve Gleason to receive the Medal of Freedom award

They met with congressmen and their staff including:

  • The offices of Senators Marco Rubio and Bill Nelson and Representatives Darren Soto, Al Lawson, John Rutherford and Ted Yoho.

Were they successful?

“There is no way to know for sure but based on questions asked by the people we met with and the notes taken we felt our mission was successful,” Paul said.

While getting support for “Right to Try” wasn’t officially part of the mission, they did discuss the topic with many of the staffers they met. Representatives Lawson, Rutherford and Yoho all voted in favor of the legislation a few weeks later.

Going forward, Rich and Paul plan to follow up with the congressmen (and their staff) to make sure their asks are met.

Want to get involved with ALS advocacy? On PatientsLikeMe, more than nearly 700 members of your community say they’re interested in advocacy — Join today to connect with others who want to make a difference.

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Want to know more about Radicava (edaravone)? Here’s a snapshot:

Posted 12 months ago by

With the recent FDA approval of Radicava (edaravone), we wanted to know more about how it works and what it means for patients living with ALS. We asked Maria Lowe, Pharm.D., BCPS, and our Health Data Integrity team, to give us a snapshot of the drug, how it’s used and what you should know. Maria’s rundown is meant to give you more context so you can have better conversations with your care team – as always, talk with your physician before starting any type of new treatment.

Radicava: The quick hits

  • Radicava works as a free radical scavenger.
  • When free radical (toxic by-products of cells that are highly unstable and reactive) levels are too high, cells are damaged resulting in oxidative stress (which may damage motor neurons). The way in which Radicava works to help patients with ALS is not fully understood yet; however, researchers believe that by getting rid of these free radicals it can help prevent some cell damage.
  • In clinical trials, treatment with Radicava was found to slow the decline in functional disability as measured by ALSFRS-r scores for some patients. However, it is important to note that Radicava does not stop the death of motor neurons (it’s not a cure).
  • Radicava may be taken in conjunction with riluzole (consult healthcare provider).
  • Radicava is administered intravenously (60 mg IV over 60 min) daily for 14 days, followed by a 14-day drug-free period. Subsequent cycles are administered daily for 10 days over a 14-day period followed by 14-day drug-free periods. Because this drug is meant for long term use, patients will need to have a peripherally inserted central catheter (PICC line) or some other kind of catheter installed.

Clinical trials and FDA approval

  • FDA approval for Radicava was based on a Phase III clinical trial conducted in Japan only in Japanese patients. Patients enrolled had to have ALS for less than two years, have normal respiratory function as well as the ability to complete most activities of daily living.
    • Radicava may be processed and function differently in different patient populations. Post marketing data will be important for determining the effects and impact of the drug in patients other than those who participated in the clinical trials.

Side effects and reactions:

  • The most common side effects reported in clinical trials include bruising, problems walking (gait disturbance), and headache.
  • Hypersensitivity reactions:  Radicava may cause allergic reactions that could happen after the infusion has finished.
    • Symptoms to watch for: Hives, swelling, dizziness, wheezing, shortness of breath, itching, and fainting.
    • If you’ve experience allergies to other medications before, inform your healthcare provider.
  • Sulfite allergic reactions: Since Radicava includes sodium bisulfite as an ingredient, those with sulfite allergies should be cautious when taking this drug.
    • Those with asthma have a higher risk of developing sulfite sensitivity. Let your healthcare provider know if you have asthma.
    • Symptoms to watch for: Hives, trouble breathing/ swallowing, itching, swelling, dizziness, asthma attacks (in people with asthma), wheezing and fainting.
    • If you have a sulfa allergy, you may or may not have a reaction to this drug. Sulfa allergies are a result of hypersensitivity to a chemical structure called sulfonamides, which are common in many antibiotics. Sulfites are chemically different from sulfonamides and any reaction to sulfites is not related to sensitivity to sulfa drugs. Both can cause reactions, but they are not related.
  • If you are pregnant or plan on becoming pregnant and/or are breastfeeding, let your healthcare provider know. It is not known if Radicava will harm the baby or if it passes into breast milk.

Availability and access

  • Radicava is expected to be available in August of this year as a brand name drug and it’s estimated it will cost around $145,000 per year in out of pocket costs (does not account for insurance coverage).
  • Because the drug isn’t available yet, many insurance companies may not have developed policies regarding coverage. Different insurance programs will develop such policies of coverage of new drugs at different times.
  • MT Pharma America (MTPA), the manufacturing company for Radicava, has developed a program called Searchlight Support to help people with ALS learn more about this treatment and to help them find and secure coverage through their insurance.
    • Searchlight Support offers out of pocket support options only to certain eligible patients with commercial insurance. Patients with Medicaid, Medicare and other government funded insurance may not qualify.

Interested in learning more about whether Radicava is right for you?

Check out the medication guide and contact the Searchlight Support program to learn more about the potential financial support programs and to find an infusion center near you.

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Disclaimer: At PatientsLikeMe, we’re all about transparency, so please keep in mind that this piece is solely to provide a snapshot of Radicava so you can stay informed and make better decisions with your care team. This content isn’t sponsored and we don’t have a financial relationship with the drug manufacturer.