FightMND Awards Grant to Lauren Sciences to Develop Promising ALS Therapy
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The Australian non-profit organization FightMND has awarded Lauren Sciences a AU$1 million ($710,000 US) grant to further the biotechnology company’s development of a new treatment for amyotrophic lateral sclerosis (ALS).
Specifically, the New York-based biotechnology company will use the funds to advance LAUR-301, a medicine that aims to reverse the progressive neurological disorder, rather than just postpone its progression. No such treatment currently exists.
Lauren Sciences specializes in medicines for neurodegenerative disorders using its V-Smart drug delivery platform. LAUR-301 is a V-Smart nanomedicine that targets brain areas affected by motor neuron death in ALS.
The pre-clinical therapy is crafted to cross the blood-brain barrier (BBB) by non-invasive administration, then to selectively deliver agents into brain areas affected by ALS degeneration, according to the company website.
It’s customized with therapeutic agent glial cell derived neurotrophic factor (GDNF), a neurotrophic protein that cannot cross the barrier by itself, which has been shown to protect motor neurons and help preserve the neuromuscular junctions — the contact point between a motor nerve cell and a muscle fiber. It has improved outcomes of ALS animal models.
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The grant project will study the therapeutic benefit of LAUR-301 in an ALS mouse model. The hope is that the U.S. Food and Drug Administration (FDA) ultimately will approve Lauren Sciences’ priority therapy.
”We commend FightMND for sharing our vision and commitment to LAUR-301,” Susan Rosenbaum, chairman and CEO of Lauren Sciences, said in a press release. “The FightMND award confirms LAUR-301’s success to date, ability to overcome the BBB [blood-brain barrier] delivery challenge, and value as a potentially life-saving therapeutic for all ALS patients.”
For its part, FightMND is excited about the potential treatment’s prospects.
”LAUR-301 has promise as an effective new treatment for ALS patients,” said Irwin Hollander, PhD, vice president of the organization’s research and development. “LAUR-301 will likely be a disease-modifying drug, unlike standard treatments that, in most patients, merely delay disease progression.”
ALS affects motor neurons in the brain and spinal cord, ultimately causing the loss of the ability to initiate and control muscle movement. According to the press release, more than 2,000 Australian residents live with ALS (also known as motor neuron disease). It also said that, based on population studies, some 5,600 U.S. residents are diagnosed with the disease annually.
The goal of FightMND is to find effective treatments and eventually a cure for the disease. The organization is the largest independent funding source of motor neuron disease in Australia.
NAFEN LIBZO
do you accept patient with ALs to take part of the trial for this drug“LAUR-301, as my sister who ai 42 diagnosed with ALS in Canda and desperate to enter any of those trial please.
my e-mail : [email protected].
BW.
nafen
B nemeth
Will this help with PLS?
B nemeth
MRI focused ultrasound opens the blood brain barrier as proven by sunnybrooke hospital in Toronto Canada
Karen
Will this trial be available to U.S. citizens via remote access?k
Crissy L Cobain
What Time frame do except to be able to use this medicine..clinical trail or after approved by the FDA?
My uncle was diagnostic on valentines of ALS. They are looking for clinical trials, but the traveling is really hard on him. Will there be a chance for the US to be in on this trial?
Thank you for your time
Manel Sarasa
My wife, diagnosed a week ago, could she be a candidate for the trial? we are in Barcelona (Catalunya)
James Hermes
I am interested in a clinical trial
Randy
can't come out soon enough.
Randy
Can't come soon enough.
dianne barret
BRAVO! please please lets get this on an international scale. I'm in France and my 35year old son has just been diagnosed. Wonderful news and gives us all hope. Thank you to you all on such an uplifting achievement for us all and congratulations on such wonderful work.
Bob Underwood
Is this expected to work with different gene mutations that cause MND? My wife is affected by C9ORF72. What was the gene mutation on the mouse model?
Thank you
James chambers
name is James chambers US Navy disabled Veteran 57 years old was diagnosed in 2016 but they misdiagnosed me since 2014 Sf VA is great hospital they found right away Reno VA neurologist maeta messed up said can't have ALS because I Walk.. I would love to be in this trial. And battle Als.
good promise this shows. Thanks again Sir.
Shkelqim Gjoni
Also my wife 40 years old is diagnosed with this terrible disease two years ago. Can my wife be a part of this trial please.
Thank you for this post!
Dave Smith
Would like to hear more on clinical trials
Sally Ann Lapka
I was diagnosed with bulbar on set in October 2018. Would this be available in the US?
Sally Ann Lapka
I was diagnosed with bulbar onset in October 2018. Will this be available in the US?
Mona Garrison
My daughter was diagnosed with bulbar onset ALS this year in May. She was misdiagnosed last Dec(2018) and is 31 years old. Can we get her into the clinical trial, please. Her name is Stephanie Polston.
Cathy
How donapply to get i to the trial? Is this available for tight to try?
Robert Briody
I'm interested in participating in a clinical trial. I'm a 74 year old Male recently and disease progression is slow. I live in Oklahoma.
Shelley Steva
Who wouldn’t want to be part of the trial?
Bucur Mihaela
Sora mea a fost diagnosticata cu SLA ,52 ani,din România. Arestează participa la studiul clinic?
MarcoFrance
Hi, it's a really good news! i'm just over 40,recently diagnose. I'm grom France, and ready to participate. For my child, his Papa is a superhero and can't give up. Help me to kill the beast.
Thanks for this very cool news!
Dale Dalessandro
My wife was diagnosed on April 3, 2019. She is 65 years old. How can we learn about enrolling for consideration to participate in the study?
Barbara McLean
Why do you tease us with the information? All the above have asked the same question. You can see we are desparate. We are living on a limited time schedule. Why not include information about where we can research if we are good candidate, where the location sites are, what the restrictions are? I know you are trying to be helpful but we struggle just to live each day. We have to jump through hoops to get assistance for our needs. We have spend hours trying to make sense of what research is out there. We have to make multiple calls to get our records sent for various things. No one can tell us what supplements to take or what exercise to do. We are told research is underfunded. We have raised millions of dollars for research. Our bank accounts are underfunded too with medical bills, medical equipment, medicines, with assistance for things we cannot do. This disease was diagnosis in 1800's. I was on a website yesterday and had to get off because every other post was about someone dying. Someone dies every 90 minutes. I want my happy ever after to see my sons get married, to be around long enough to see my grandchildren. What is it going to take?
Mona Garrison
My daughter was diagnosed with bulbar onset ALS this year in May. She was misdiagnosed last Dec(2018) and is 31 years old. Can we get her into the clinical trial, please. Her name is Stephanie Polston.
Anna Omsberg
I am interested in information as well. My mom, 56, was diagnosed with ALS last week. She still walks and talks but her foot is hard for her to pick up. We have started Whole 30 diet and take multivitamins to detox. Any information is appreciated.