Posts Tagged ALS
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.
Hard 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; Neil 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 :).
This 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 .
Posted by Darshana V. Nadkarni, Ph.D. in Biotech - Medical Device - Life Science - Healthcare on August 4, 2014
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.
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.
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.