To paraphrase Newton's law of inertia, a blogger persists in a state of rest unless acted upon by an external force (of will power). I have been rather negligent in passing along to the few who read or happen to come across this blog the latest in MJD research.
Here are some very encouraging news from the scientific front-lines, thanks to the MJD Family Board for sharing the information:
RNAi has potential for use in MJD treatment and constitute the first proof-of-principle for allele-specific silencing in the central nervous system
More about the aforementioned research
CATENA® : a new drug approved for the symptomatic management of patients with Friedreich’s Ataxia which may be beneficial in treating MJD
and lastly
Geron Receives FDA Clearance To Begin World's First Human Clinical Trial Of Embryonic Stem Cell-Based Therapy
On the home front, Richard's hard work at his twice weekly PT is paying off. Today, he was able to go from a sitting position to standing upright unassisted three consecutive times without fumbling or tottering. His movements when using the walker are steadier and more fluid. Small steps but important steps which is all that we are asking for.
Tuesday, January 27, 2009
Tuesday, January 6, 2009
The Curious Case of the Naked Man on the Floor
I've decided to begin the new year with a more light-hearted post. A few days ago, while researching the latest on stem cells on the web, I came across a set of articles (1, 2, 3)from the Charleston Post and Courier about a patient who was at Nanshan Hospital around the same time as we were.
Upon reading the article, I realized that the gentleman in the articles was the same person that I described in my post dated March 27, 2007. These news articles helped me understand better his experience in China. While the hospital staff and the reporter each painted a different picture of this patient's personality and deeds, I am sure that he meant to do no harm. Furthermore, I empathize with his desire and need to do everything within his power to get better.
The famous movie producer, Robert Evans, once said, "There are three sides to every story. Your side, my side and the truth. And no one is lying." I couldn't have put it better myself.
Upon reading the article, I realized that the gentleman in the articles was the same person that I described in my post dated March 27, 2007. These news articles helped me understand better his experience in China. While the hospital staff and the reporter each painted a different picture of this patient's personality and deeds, I am sure that he meant to do no harm. Furthermore, I empathize with his desire and need to do everything within his power to get better.
The famous movie producer, Robert Evans, once said, "There are three sides to every story. Your side, my side and the truth. And no one is lying." I couldn't have put it better myself.
Sunday, November 23, 2008
It was the best of times, it was the worst of times
2008 has been a momentous year so far, filled with both memorable celebrations and challenges that we'd rather do without.
Richard and I celebrated our 25th wedding anniversary and half century birthdays. We also reunited with family members who we have not seen for some time and participated in Richard's mother's 80th birthday bash in New York.
However, our mothers' health problems continue to plague them. Richard also had his share of health issues in the past few months. There were falls and accidents that resulted in trips to the emergency room. The numerous stitches on his face has given him a "tough guy" aura.
Looking beyond our immediate family, we now have a charismatic African-American president-elect who is poised to bring much needed change to the nation. However, the economy has plunged into unprecedented chaos that threatens the livelihood of every global citizen.
It is hard to remain positive in these difficult times. I take comfort in Nietzche's quote of "that does not kill us makes us stronger".
Richard and I celebrated our 25th wedding anniversary and half century birthdays. We also reunited with family members who we have not seen for some time and participated in Richard's mother's 80th birthday bash in New York.
However, our mothers' health problems continue to plague them. Richard also had his share of health issues in the past few months. There were falls and accidents that resulted in trips to the emergency room. The numerous stitches on his face has given him a "tough guy" aura.
Looking beyond our immediate family, we now have a charismatic African-American president-elect who is poised to bring much needed change to the nation. However, the economy has plunged into unprecedented chaos that threatens the livelihood of every global citizen.
It is hard to remain positive in these difficult times. I take comfort in Nietzche's quote of "that does not kill us makes us stronger".
Sunday, October 12, 2008
More from ACARM7
We saw Dr. Perlman recently when Richard had his semi annual checkup with her at UCLA. I asked Dr. Perlman for a copy of her ACARM7 talk since I was only able to summarize a small portion of her presentation in my last post. Aside from news of the latest trials for HD and SCAs, it has pointers and information germane to the SCAs.
Mazel Tov to Dr. Perlman who was presented with the prestigious Sherman M. Mellinkoff Faculty Award this past May. The award is considered the highest faculty distinction at the UCLA Medical School.
Mazel Tov to Dr. Perlman who was presented with the prestigious Sherman M. Mellinkoff Faculty Award this past May. The award is considered the highest faculty distinction at the UCLA Medical School.
Sunday, September 14, 2008
Notes from ACARM7
We just came back from the 7th All California Ataxia Research Meeting (ACARM7) held at the San Jose Doubletree Hotel today. This year's meeting was almost canceled due to budget issues stemming from lower than expected attendance. At the eleventh hour, it was saved by a generous anonymous underwriter. The other unsung heroes are Mike Fernandes, the organizer of the venue, the doctors and presenters of the program and the numerous volunteers who helped to bring the event into fruition.
The first speaker of the morning was Dr. Richard Ivry who talked about "The Effect of Ataxia on Cognition". Specifically, he spoke about Dr. Jeremy Schmahmann's work on the "Cerebellar Cognitive Affective Syndrome". Dr. Ivry concluded that ataxics have difficulty with non-motor tasks but their cognitive problems might be secondary to their motor problems.
Dr. Susan Perlman was next. Her topic was "An Update of Clinical Trials for the SCAs and HD". According to Dr. Perlman, if one were to do a web search on the key words "clinical trials for Ataxia", one would find 42 trial listings of which only 35 actually dealt with Ataxia. 13 of these trials have been completed, 5 are active but no longer recruiting for subjects while the remaining 17 are still recruiting. Of these 17, one is available in California ---- Phase III Idebenone and Extension Study for FA. (Friedreich's Ataxia) Also, the pilot study of Lithium for patients with SCA1 has now started in Maryland and is still recruiting.
Dr. Lisa Ellerby followed Dr. Perlman with "Target Validation in HD: siRNA screens in HD cell culture models". After conducting studies to screen through possible druggable targets using siRNA technology, her team has found that HDAC4 inhibitors to be effective against Huntington's Disease.
Dr. Elizabeth Thomas was the last speaker of the morning. She further elaborated on the HDACs with "Novel Histone Deacetylase Inhibitors as Therapeutics for Triplet Repeat Disorders". While conventional HDAC inhibitors (used in treating cancers) are very toxic, novel HDAC inhibitors have been shown in (non-human) trials to be low in toxicity. Experiments on transgenic mice using HDACi4b have shown it to be therapeutic and non toxic for triplet repeat disorders such as HD, SCA1, 2 and 3. Repligen Corp. in Waltham, Massachusetts has licensed HDAC inhibitors in development.
The three presenters in the afternoon were Richard Devylder, (Emergency Disasters for the Disabled) Colleen Campbell (Falling Prevention) and Arya Pathria. (Laughing: Laughter Yoga Workshop)
The first speaker of the morning was Dr. Richard Ivry who talked about "The Effect of Ataxia on Cognition". Specifically, he spoke about Dr. Jeremy Schmahmann's work on the "Cerebellar Cognitive Affective Syndrome". Dr. Ivry concluded that ataxics have difficulty with non-motor tasks but their cognitive problems might be secondary to their motor problems.
Dr. Susan Perlman was next. Her topic was "An Update of Clinical Trials for the SCAs and HD". According to Dr. Perlman, if one were to do a web search on the key words "clinical trials for Ataxia", one would find 42 trial listings of which only 35 actually dealt with Ataxia. 13 of these trials have been completed, 5 are active but no longer recruiting for subjects while the remaining 17 are still recruiting. Of these 17, one is available in California ---- Phase III Idebenone and Extension Study for FA. (Friedreich's Ataxia) Also, the pilot study of Lithium for patients with SCA1 has now started in Maryland and is still recruiting.
Dr. Lisa Ellerby followed Dr. Perlman with "Target Validation in HD: siRNA screens in HD cell culture models". After conducting studies to screen through possible druggable targets using siRNA technology, her team has found that HDAC4 inhibitors to be effective against Huntington's Disease.
Dr. Elizabeth Thomas was the last speaker of the morning. She further elaborated on the HDACs with "Novel Histone Deacetylase Inhibitors as Therapeutics for Triplet Repeat Disorders". While conventional HDAC inhibitors (used in treating cancers) are very toxic, novel HDAC inhibitors have been shown in (non-human) trials to be low in toxicity. Experiments on transgenic mice using HDACi4b have shown it to be therapeutic and non toxic for triplet repeat disorders such as HD, SCA1, 2 and 3. Repligen Corp. in Waltham, Massachusetts has licensed HDAC inhibitors in development.
The three presenters in the afternoon were Richard Devylder, (Emergency Disasters for the Disabled) Colleen Campbell (Falling Prevention) and Arya Pathria. (Laughing: Laughter Yoga Workshop)
Friday, August 1, 2008
Finding your inner Tigger
Just in case you missed "The Last Lecture", I highly recommend it on those "why me" days.
Wednesday, July 9, 2008
All California Ataxia Research Meeting 2008 (ACARM7)
ACARM7 will be held on Sunday, September 14, 2008 at the Doubletree Hotel by the San Jose Airport. This year's meeting will concentrate on the SCAs. Richard and I are looking forward to the event. As usual, kudos to Mike Fernandes for putting this together.
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