Thursday, January 31, 2013

Deer Antler Velvet?

A major issue with those of us living with a disease that does not have a treatment or cure is some of us become desperate. We read about something that works for another similar disease, or is advertised that it works in building muscle, and we think, “Why not give it a try?”

Well, there are plenty of reasons to ‘not give it a try’. The drug or concoction is not always safe or proven to work. It could also be dangerous, especially if we are not aware of the dosage. Finally, what other problems (side effects) might it cause?

deer antlerThe last few years IGF-1 for muscles and ASC-J9 are two drugs that have been discussed by researchers as having a potential benefit for Kennedy’s Disease (SBMA or Spinal Bulbar Muscular Atrophy). They are both currently being tested with small mammals. One of the big issues with both drugs is how to decide upon a dosage that will work, but will do no harm. And, if it does work, another issue is how to make the drug available (injection, pill, IV, etc.) and convenient.
Another issue with those of us living with Kennedy’s Disease concerns the mutation in our DNA. Some drugs will not help, and could possibly do more harm, because of this defect.

IGF-1Recently, several articles have been published about athletes taking a banned substance called IGF-1 or a supplement made from deer antler velvet. The athletes are using the drug to improve their strength (in other words, cheating). Several people with Kennedy’s Disease have asked about using IGF-1 that you can by over the internet or in a foreign country.

We consulted with a member of the KDA’s Scientific Review Board concerning the IGF-1 you can buy over the internet.  We received the following reply:  “I do not recommend the sublingual IGF-1 advertised on the internet site you sent. The IGF-1 (being test) in mice and the IGF-1 currently approved for human use are only available by injection, and neither has yet been shown to be safe and effective in KD patients.”

Being someone who is not getting any younger, I understand the lure of a potential treatment for Kennedy’s Disease. I also realize that patience is a virtue. NIH is testing both the drugs mentioned above. Hopefully there will be a clinical trial for one or both in the next couple of years. If and when it does become available for human use, it will be safe and effective.


Wednesday, January 23, 2013

On my way back

exerciseI have been off Warfarin for three days now. I began to notice a difference yesterday. This morning when I began my standing exercises the difference was amazing. I did 24 calf steps, 30 side-steps and 60 step ups without any problems. The weakness in my trunk muscles (stomach and back) was also gone. Just two days ago this was impossible. I went on to perform all of my other long-day exercises. I noticed that some exercises were still not perfect, but they were a lot easier and better.

Another difference I noticed yesterday was the heaviness I felt in my lungs was gone.

Observation: It wasn’t the flu that caused this extreme weakness.  I must be one of those 5% that cannot take Warfarin because it causes muscle weakness and fluid on the lungs.

In two more days I begin a new blood thinner. Two of the benefits of this new thinner are that I do not need INR blood tests and I can eat any food item (salads, broccoli, cranberries, etc.). I am really enjoying eating salads again. In the expansive potential side effects list is one that says, “Could cause muscle weakness.” So, I need to be observant and make certain this doesn’t happen again.

Sunday, January 20, 2013

Something has to change

I thought I was recovering well from the flu, but for some reason my strength has not returned. Even worse it seems to be declining. I had a few decent days early in last week, but the last three days have been very difficult. Because of this weakness in my left leg and right arm, I have lost confidence in my ability to stand. Also, my trunk strength (abdomen and back) is very weak adding to my difficulties.

weaknessWhere I normally perform 36 calf stretches, 60 side steps, 100 step ups, and 18 leg swings, I find it difficult to just do 20 side steps. Then I have to rest. Then, I can maybe do 20 step ups. If I wait long enough, I can do 18 calf stretches. I still haven’t been able to do any leg swings.

Is the weakness related to the flu or something else? Could it be related to the Warfinin?  I need to make an appointment with my doctor to see what is happening. Perhaps he can figure out why my strength is not returning. All I know is that something has to change because the current situation is not manageable. 

Living with Kennedy’s Disease is difficult (especially as we get older), but this sudden and unexplained loss of strength is something unexpected.  I need answers.

Thursday, January 10, 2013

One of the 40%

The CDC commented that 40% of those who had the flu shot this year will still get the flu.

fluYup, I had my flu shot and I still got the flu. And, just as I always warned, those of us living with Kennedy's Disease have to be extra careful when it comes to the flu.

It is day eleven and I feel between 80-to-90% well. I still have the cough, but not quite as bad. The fever and aches are gone. Day two and three were the worst … just plain miserable. I ended up sleeping most of the day (between coughs anyway). I have been extra cautious since coming down with it and very mindful of the seriousness of this health issue should it turn into pneumonia.

The weakness is still there, but I am improving every day. I have exercised for 50-to-75 minutes the last five days and that seems to help (as well as raise my spirits). I have always said ‘patience is a virtue’. It is also an attribute that I need to develop further. Having the flu this year has been a test for me.

I still highly recommend the flu shot and the pneumonia vaccine when you turn 65. I still believe we have to be extra careful this time of year. I was at the doctor’s office once a week for eight weeks having my blood tested (INR). If I hadn’t been exposing myself weekly I would probably have been safe.

The only real benefit to having the flu this time of year is all the collegefootball-bowls bowl games. I never felt guilty sitting around and watching every game even though I often fell asleep halfway through some of them.

Friday, January 4, 2013

30th Anniversary of the Orphan Drug Act

nord-member-org.300x100Most of you know I am an advocate of NORD (National Organization for Rare Disorders).  They have done a lot of good representing those of us living with rare disorders including Kennedy’s Disease.  This year is the 30th anniversary  of the Orphan Drug Act.  Below is a press release the KDA received today.

NORD Press Release


Launching a Year-Long Celebration: 30th Anniversary of the Orphan Drug Act and NORD

Washington DC, Jan. 4, 2013----Thirty years ago today, President Ronald Reagan signed the Orphan Drug Act into law to encourage the development of treatments for the millions of Americans with rare diseases.

The new law represented a triumph of patient advocates working with government partners, the media, and other supporters to address a critically important unmet need.

To get the law passed, an ad hoc coalition of leaders of rare disease patient organizations gave themselves a name -- the National Organization for Rare Disorders (NORD) -- and taught themselves how to be advocates for the desperate patients and families they represented.

Actor Jack Klugman, whose brother was dying of a rare disease, lent his support with an episode of his popular TV show, Quincy, M.E., dedicated to showing the need for treatments for rare disease patients.  Klugman later testified before a Congressional subcommittee and, when the bill became stalled in Congress, aired another Quincy episode that helped get the bill passed.

Exactly four months after President Reagan signed the bill into law, on May 4, 1983, the coalition of patient advocates formally established NORD as a nonprofit organization to provide advocacy, education, research and patient/family services for all Americans affected by rare diseases.

"It was an amazing accomplishment to get this desperately needed law enacted at that time," says NORD President and CEO Peter L. Saltonstall.  "Today and throughout 2013, we will be saluting all the pioneers who helped make it happen, celebrating progress to date, and working with our partners to find ways to accelerate the pace of progress."

Saltonstall added that the recent death of Jack Klugman (on Dec. 24th) added poignancy to the occasion.

NORD's president for its first 25 years, Abbey S. Meyers, is widely considered the primary patient advocate for the Orphan Drug Act.  She retired in 2008 and Saltonstall joined the organization at that time.

Government partners at the National Institutes of Health (NIH) and Food and Drug Administration (FDA) have provided major support and encouragement to the rare disease community through their implementation of the law.

On Monday, the FDA Office of Orphan Products Development will host an event for FDA staff and guests to observe the anniversary of the law.  FDA Commissioner Margaret Hamburg, MD, will speak, as will Saltonstall, with a video message from Meyers.  Special guests will include individuals and representatives of organizations being honored as "rare disease heroes" by FDA during the anniversary year.

NORD's year-long observance of the 30th anniversary will include a special section on its website where rare disease patients, researchers, and others will be invited to share stories about rare disease milestones and achievements.

On Rare Disease Day 2013 (Feb. 28th), which NORD sponsors nationally, there will be special activities to honor the history of the rare disease community.  In addition, NORD's annual gala in May and annual conference in October will have an anniversary theme.  There will be other special activities throughout the year.

The Orphan Drug Act provides financial incentives such as seven years of marketing exclusivity for products developed to treat diseases affecting fewer than 200,000 Americans.  There are 7,000 diseases considered rare in the U.S. affecting nearly 30 million Americans, or about 1 in 10 people.
In the decade before the law was passed, only 10 new drugs were developed by industry for patients with rare diseases.  Since 1983, more than 2,700 potential treatments have entered the research pipeline as "orphan products" and more than 400 have been approved by FDA.

Highlights over the years since the law was enacted have included the Rare Diseases Act of 2002, promoted by NORD, which made possible a Rare Diseases Clinical Research Network at NIH, and most recently the FDA Safety and Innovation Act of 2012, which includes many major provisions related to rare diseases.

"This year, we will be saluting the progress made to date but we'll also be focusing on the fact that much remains to be done," Saltonstall said.  "Most people with rare diseases still have no FDA-approved treatment.  And, even when treatments exist, many patients have trouble accessing them.

"During this anniversary year, NORD will move forward with renewed commitment toward the development of safe, effective treatments for all, and assurance that all patients with serious medical problems will have access to the treatments and services they need."