Posts Tagged ALS

2014 – Year-end Review ——- Theme: Confront Reality & Get Things Done!


English: Biosafety level 4 hazmat suit: resear...

English: Biosafety level 4 hazmat suit: researcher is working with the Ebola virus (Photo credit: Wikipedia)

Year 2014 is coming to a close.  As I see it, it has been a year to confront the reality and get things done.  Affordable Care Act became law in January, increasing the accessibility of healthcare, in the US.  TIME declared Ebola as the defining issue of the 20th century. It was no more in remote regions of Africa but in the capitals and it landed in the USA.  The reality is that we are living in a global world.  

And then President Obama reminded us that even though some of our neighbors entered the border illegally, they have made their home here, are working hard, supporting their families and they should have NOT amnesty, but an opportunity to make it right with the law, and live here temporarily, AND pay their share of the taxes, because people can’t live in the shadows, in a global world of visibility and accountability.   Long overdue immigration reform will enable many people to come out of the shadows and add to the national treasury – a win-win – what’s not to like?

The same applies to our gay neighbors.  They should not have to live in the shadows.  Majority of the states this year, legalized same sex marriages and US supreme court refused to hear appeals from states seeking to keep same sex marriage ban in place.  Many states also legalized marijuana.  But what about our veterans living in the shadows?  Department of Veterans Affairs got more resources (as house passed the bill, at the end of the year, averting shutdown), and it now has to get its act together and make it right with those who defend our freedom and values.

And what about skin color?  We are confronting the reality that more than 50 years after Dr. King laid out his vision for color-fair society, people are still being judged on the basis of the color of their skin and paying with their lives.  This does not just happen when young men turn 22 but prejudice hits in childhood  http://bit.ly/15EInJ4 and it splinters society.  We can heal and move ahead, but scars made by history, and distrust can only heal when there are no new wounds, when there is real dialog, when each side gets to even briefly experience the reality that the other lives with, and have compassion.  We are confronting the reality and lot of dialog is happening.  Much work remains to be done but the issue can’t be ignored any longer.

And then the lowest of the low, terrorists and those plotting terror.  How do we deal with them?  Report on CIA’s use of “enhanced interrogation techniques” in the post-9/11 era reveals that “CIA detainees were tortured”.  When we lose sight of our values, when the boundaries between moral and immoral gets blurry, then we lose, regardless of what we were seeking to gain.  This is a true moment for national soul searching. (the fact some people may be only suspected of being terrorists is whole other story).

Globally, also we are confronting realities.  World’s largest democracy, India, elected controversial Mr. Narendra Modi as Prime Minister.  Mr. Modi has reached out to leaders across the world and declared campaigns to clean up India.  My birthplace has so much to offer to the world and if it cleans up its act, under the helm of Mr. Modi, I couldn’t be happier.  Our neighbors need to clean up their act too, even as they rightfully blame the US for its insatiable appetite for drugs.  Capture of “El Chapo” Guzman in Mexico was a HUGE victory that got overshadowed later by disappearance and ruthless murder of 43 Mexican college students.  “#YaMeCanse12”!  Abduction of 270 high school girls by Boko Haram in Nigeria #BringBackOurGirls and scores of Yazidi women bought, sold, raped, and murdered, underscores the need to define rape during conflict as a war crime and not a woman’s issue.  By some estimates, more than 7 million (50% are children) are displaced by war in Syria (200,000 are killed) and 100,000+ Yazidis are displaced by ISIS.  Let us continue to keep theses issues in the spotlight.

The year is ending on a rather sad note of the children who lost their lives in #PeshawarSchoolAttack in Pakistan.  Here is my short poem in their memory – http://bit.ly/1wfp47D .  It was heart warming to see India support its neighbor in the hour of grief as #IndiawithPakistan was a popular hashtag on twitter. And also deeply heartwarming to see Pakistan echo the sentiments when #PakistanwithIndia and sepecially #PakistanwithIndiaNoToLakhviBail became trendy topics on twitter as overwhelmingly Pakistani people reacted negatively to their government’s decision to give bail to Mumbai terror mastermind Lakhvi.  May the balanced sentiments always prevail over extremism, because the reality is that we live in a global world and terror can’t be nurtured and targeted because sooner or later it would hit home.  Global world also demands secularism.

As a ray of light and hope, Malala Yousafzai, courageous young lady from Pakistan, spearheading girls’ right to education and Kailash Satyarthi from india, a brave and dedicated activist for children’s rights and against child labor, shared the Nobel Prize, sending strong messages that fight to honor children’s rights will continue.

Personal

wpid-20140920_173123.jpgHard as it is to confront the reality that one’s parents may not be there forever, I was very happy to spend wonderful time with my mother and my aunty (her sister).  I tried to focus on giving them a break from their routines and enable them to have some fun, some unusual experiences.  Isn’t it amazing that when a mother gives, she gives with her heart and soul, but when she receives from her children, she receives with a feeling of enormous debt and gratitude!  Both my children are focused on their careers; wpid-20140805_201145-1.jpgNeil is working with Cisco in IT and Neesha is finishing college this coming year.  Both are my pride and joy :).  It has also been fun hosting my daughter’s friend from UCSD, originally from Palestine, during the holidays, and alternately being “naughty” with the girls, and playing aunty-mom to two daughters :).wpid-20141221_150415.jpg

wpid-20141221_161442.jpgThis year, I also visited Japan (we were hosted by many amazing friends and you can see all details in my travel blogs), an amazingly polite and most efficient culture, with world’s most interesting toilets http://bit.ly/1sYL5qs.  This year I also started travel blogs and you can see my many blogs at www.darshanavnadkarni.wordpress.com.

And finally, here are links to some of the most amazing things that I blogged about, this year.
Best movie – “Last Days in Vietnam” http://bit.ly/1qFIL28
Best play – “Truce” http://bit.ly/1trGhEG and “Andhera Hone Tak” http://bit.ly/1Aij5Rz
Best book – “The Glass Castle” http://bit.ly/1fchcIo
Best biomedical technologies — so many exciting technologies in early to mid stages of development for — treatment of ALS http://bit.ly/1AP2Yd0, for technology for early detection of cervical cancer http://bit.ly/1jalqEz, technology that aims to deliver drugs via inhalation for AFib, point of care solution to minimize prescription filling errors http://bit.ly/1jdfmgr

Wishing my readers, family & friends, and my clients and colleagues, peace and joy in the year 2015.  Best wishes to my many friends in fantastic groups that I am routinely affiliated with (each of them enhance life for many, personally and/or professionally) http://www.bio2devicegroup.org, http://www.eppicglobal.org, http://www.citylights.org, http://www.thestage.org, http://www.theatreworks.org, http://www.naatak.org, http://www.enacte.org, http://www.iwings.org .

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“The Theory of Everything” – Movie Review


In 1960s, a brilliant young man introduces himself as a cosmologist, to the girl who later becomes his wife.  When she asks what it means, he says, “it is a kind of religion for intelligent thinkers”.  And there begins the true story of one of the most brilliant thinkers in history, Stephen Hawking (Eddie Redmanyne) and his sweetheart, wife and mother of his children, Jane Wilde (Felicity Jones).

Right before Jane and Stephen got married, Hawking was found to have motor neuron disease (also called amyotrophic lateral sclerosis or ALS or Lou Gehrig’s Disease), and received prognosis of two year survival (see my article bit.ly/1AP2Yd0  on possible novel treatment for ALS). Jane married Stephen in 1963, despite being warned that the “weight of science was against her”.  In a race against time, Stephen, with Jane’s help, started working on science.  Stephen is miraculously still alive today and is in his 70s.  In their life’s journey, Stephen and Jane encountered many trials and tribulations, became parents of three children and are now grandparents.  They married, they loved deeply, they eventually divorced, and they continue to remain friends.

English: Stephen Hawking giving a lecture for ...

English: Stephen Hawking giving a lecture for NASA’s 50th anniversary (Photo credit: Wikipedia)

Interwoven with the great love story is the story of science which began with Hawking’s search of a “single unifying equation that explains everything in the universe”.  In his earlier work, Hawking developed detailed mathematical models to explain the property of black holes from which nothing escaped.  But later, explaining from combined general theory of relativity and quantum mechanics, he opined that black holes are not black, after all.  Hawking explained that these so called black holes glow with “heat and radiation and eventually everything blows up in a spectacular explosion”.

Jane explains Quantum theory, versus Relativity theory with the help of peas and potatoes, to Jonathan Hellyer Jones (Charlie Cox), a family friend.  Hawking had begun revising his thinking.  He had always been an atheist and in the conversation with Jonathan, Hawking explains, quantum theory by itself was falling short in the explanation, “so God is once again on endangered species list and physics is back in business”.  With his “no boundary theory”, Hawking explains in his new book “The Brief History of Time”, that time and space would have no boundary or edge and the laws of science would hold everywhere, including at the beginning of the universe.

Jonathan becomes a close family friend and eventually, he and Jane develop feelings for each other.  Also Hawking develops a new relationship with his therapist, Elaine (Maxine Peake).  During a visit to Geneva, Stephen contracted pneumonia and only survived with a tracheotomy, which also resulted in him losing his speech entirely.  Even though Stephen and Jane go through many challenges that take a toll on their 25 year old marriage, it is clear that their relationship was not only built on deep emotional love but it was a meeting of the minds; their conversations provided intellectual stimulation and they got creative insights from togetherness, in their space and time.  This movie, adapted from Jane Hawking’s book, “Travelling to Infinity: My Life with Stephen”, celebrates their incredible relationship.

Hawking is indeed a remarkable human being.  He is incredibly brilliant and he has defied all odds and beaten all predictions made about his disease and quality of life.  More importantly, the woman who stood by his side and enabled his most meaningful contributions in the field of science, also added richness and meaning in his personal life.  Although Hawking continues to remain an atheist, he seems to have become mellower and more reflective.  Recently , he mentioned through social media that he was moved by watching the movie and it gave him an “opportunity to reflect” on his life.  In the movie, towards the end, Hawking was asked if he had a philosophy for life and he replied, “just as universe has no boundary, there is also no boundary to human endeavor; where there is life, there is hope.

Director James Marsh has done a brilliant job of capturing the complexity of Jane and Stephen Hawking’s beautiful relationship, of Stephen’s disabling body, and a brilliant mind, of wit and humor with which he approaches life.  The movie does not overplay the pitiable aspects of the devastating disease and balances the shadow of the disease in Stephen and Jane’s relationship, with love and romance in their life. From a young man in love, to a man who is slightly clumsy at the onset of ALS, to a man who ages, whose marriage is getting old, and who struggles through the progression of the deadly disease, Radmanye, who lost 15 lbs. for the role, met 40 ALS patients, trained with a dancer to control his body, and stood in front of the mirror for hours contorting his face, gives an absolutely incredible performance.

And while the movie focuses on the everyday aspects of Stephen and Jane Hawkins’ life, family, and children, it also provides some powerful metaphors (e.g. peas and potatoes, wind back the clock), so characteristic of Hawking, to shed light on his theories.  Unfortunately, the film only provides a drop from the vast sea of science produced by Hawking.  What I would have loved to see is more science and more explanation of significance of Hawking’s contributions.  This still remains a fantastic movie about the life of two amazing people and on my scale of 1-5, with 5 being excellent, I give it a rating of 4.8.

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Treating ALS – The Story of Neuraltus


Rich Casey, President and CEO of Neuraltus (formerly Chairman and CEO of Scios), talked about treatment option for ALS (Amyotrophic Lateral Sclerosis), also known as Lou Gehrig’s Disease, a fatal neurodegenerative disorder of unknown etiology, at www.bio2devicegroup.org event.

Typically, the age of onset of ALS is anywhere between 40 and 70 years.  Life expectancy after the diagnosis is between 2 and 5 years, although in very rare cases (e.g. in case of Stephen Hawking), it could be an extremely slow progression.  No effective treatments for the disease exist.  Motor neurons degenerate and early symptoms include muscle weakness, specifically involving arms and legs, lack of hand grip, deep tiredness and it progresses to difficulty in swallowing, breathing, and garbled speech.

Macrophage

Macrophage (Photo credit: Wikipedia)

Neuraltus’s lead product,  NP001 is in clinical development, targeted for the treatment of ALS.  While the exact cause of the disease is unknown, there is fair amount of evidence that points to increasing levels of inflammation in the macrophages.  This increased inflammatory activity results in the release of factors in the central nervous system that leads to damage of motor neurons.  Macrophages are cells produced by differentiation of monocytes in the tissues.  Macrophages are highly specialized in removal of dead cells or debris.  Additionally, macrophages also “present” antigen that plays a crucial role in initiating an immune response.  There is an increasing evidence implicating neuroinflammation with the progression of ALS.

English: Amyotrophic lateral sclerosis MRI (ax...

English: Amyotrophic lateral sclerosis MRI (axial FLAIR) demonstrates increased T2 signal within the posterior part of the internal capsule, consistent with the clinical diagnosis of ALS source:Radiopedia.org (Photo credit: Wikipedia)

Neuraltus’s novel, proprietary drug, NP001 regulates macrophage activation and converts the activated inflammatory macrophages from activated, neurotoxic inflammatory state to a neuroprotective state.  It thus normalizes the critical cellular environment.   Phase I, single dose study indicated that that there are no safety concerns and the drug is well tolerated.  Further, in a Phase II safety, tolerability and preliminary efficacy study, after administration of NP001, there occurred a close dependent reduction of inflammatory macrophages.

The dosing regimen is somewhat cumbersome, said Casey.  First time, the patients come in the hospital and need to stay for 5 days and subsequently for 3 days.  At low dose, phase II study results indicated positive trends in the ability of NP001 to slow the rate of disease progression but did not reach statistical significance.  But at high dose, the drug freezes the disease in a 3rd of the patients.  Additionally, the drug is found to be safe and well tolerated.  This clearly looks like a very exciting potential treatment for a devastating disorder, ALS.  The company is currently looking for funding.  The talk was followed by Q&A.

 

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