U.S. Department of Defense Earmarks $758,121 to Develop RASRx1902 as Potential ALS Therapy

U.S. Department of Defense Earmarks $758,121 to Develop RASRx1902 as Potential ALS Therapy

The U.S. Department of Defense (DoD) has issued a two-year research grant totaling $758,121 to support a group of scientists from the University of Arizona Health Sciences Center for Innovation in Brain Science (CIBS) working on the development of RASRx1902, a potential treatment for amyotrophic lateral sclerosis (ALS).

The project, led by Kathleen Rodgers, PhD, and Kevin Gaffney, PhD, will focus on evaluating the effects of the compound in cells from ALS patients cultured in a lab dish. The main goal of these experiments is to determine first if RASRx1902 can improve the overall health of patients’ cells, and then see at which stage of the disorder the treatment is most effective.

“The goal of our research is to develop tomorrow’s cures for patients that need them today,” Gaffney, an assistant research professor at CIBS, said in a press release. “We are excited for this opportunity to assess the potential of RASRx1902 to treat ALS. We truly appreciate the DoD’s investment in this important research.”

RASRx1902 is an investigational oral drug that has been shown to reduce inflammation and oxidative stress (cellular damage that occurs as a consequence of high levels of oxidant molecules), improve cognitive function, and stimulate muscle regeneration in previous studies.

Because of its muscle regenerative properties, RASRx1902 has been explored as a potential treatment for Duchenne muscular dystrophy (DMD), a genetic disorder that gradually leads to muscle deterioration. Studies in animal models of DMD have shown the compound increased muscle strength and regeneration, reduced muscle inflammation, degeneration and cell death, without posing any toxic side effects.

Based on those promising findings, the U.S. Food and Drug Administration (FDA) granted the designation of orphan drug to RASRx1902 for the treatment of DMD in 2017.

In the new project, Rodgers and Gaffney will attempt to gather pre-clinical evidence demonstrating the effectiveness of RASRx1902 in ALS. If they are successful, RASRx1902 then may be tested in ALS patients participating in clinical trials.

“This is a critical stage of discovery in our pursuit of a cure for ALS,” said Rodgers, associate director of translational neuroscience at CIBS and principal investigator of the study.

“We have an urgent need for treatments that will have immediate and long-term benefits for individuals with this devastating disease. RASRx1902 has the potential to improve the treatment, quality of life and long-term outlook for ALS patients and their families,” Rodgers said.

Joana is currently completing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. She also holds a BSc in Biology and an MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that make up the lining of blood vessels — found in the umbilical cord of newborns.
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Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Técnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.
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Joana is currently completing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. She also holds a BSc in Biology and an MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that make up the lining of blood vessels — found in the umbilical cord of newborns.
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11 comments

  1. Dave Reckonin says:

    “The goal of our research is to develop tomorrow’s cures for patients that need them today,” Gaffney, an assistant research professor at CIBS, said in a press release.”

    A very glib and patronizing phrase.

    Not surprisingly, Gaffney omits timescales for this project, confirming what we all know to be the case that the pace of investigations remains uniformly sloth-like.

    ‘Sloth-like’ is as good as it gets these days.

  2. ReasonAndWisdomMike says:

    I wish we had more time, more time to spend with our families and friends, more time to try out new treatments, more time to spend with my son who’s only 1 year old.
    You don’t have to cure me of ALS. I just need more time please.

  3. I’m working on understanding the disease. So many paths to follow. First, it is a series of events. The question is where does the series start. For some diseases it doesn’t matter if we know where it starts or how it progresses, we can still treat and cure. Maybe understanding the series of events will help and maybe it won’t. One fellow researcher at Johns Hopkins says, ‘It must be something upstream’ referring to the fact that a chain of events leads to the muscle deterioration and loss of signalling between the brain and the muscles. Some areas of interest for my research are serotonin, dopamine, potassium, sodium,sleep deprivation, head injury, pathogens,Lyme and so many other areas to try and figure out what causes ALS and what may cure it. The biggest area of promise right now may be, for me, in understanding the role serotonin plays and whether an SSRI may help to halt or even reverse deterioration. Each of us is dependent upon dopamine for movement. Without it our bodies simply cannot move. Dopamine and serotonin walk hand in hand. Sleep is critical for the body to restore dopamine and serotonin levels and to eliminate waste products from the body’s neurotransmitter system. It’s possible that central sleep apnea – far different than obstructive sleep apnea, may cause the body to dysregulate serotonin and dopamine to the point it causes a gradual breakdown in the way the brain talks to the muscles. Once the breakdown in communication happens, toxins build up and and the process gets completely out of hand to the point we see the outward symptoms of ALS. The hope is to find out how to re-regulate the body and to quickly clean up the toxins. Our bodies are a series of non stop chemical reactions. One imbalance leads to others and eventually things break down. Prayer is the number one research tool as only God Himself knows what causes and what can cure ALS. Please pray for a cure and for researchers to look in the right places for the answers. 😊

  4. Cindy says:

    It’s good to know that some are investigating muscle regeneration. Slowing and stopping are great but reversing is best.

  5. Melissa Whipple says:

    I have ALS. First symptoms occurred in 7/16; diagnosis did not occur until 2/18.
    Of course, I had to research my disease sufficiently enough to figure out:
    -How to Qualify for any clinical trial, let alone which ones showed any likely benefit (I have sporadic ALS)
    – If they were currently recruiting, and were located within in a practical traveling distance
    -Apply–many times the criteria (which may or may not be listed) for being accepted into an ALS Clinical Trial had recently changed –such as the acceptable age of ALS onset had recently been lowered (from 65 years old-to under 60 years old) (NurOwn) or the criteria for time expired since one’s formal ALS diagnosis (or first symptoms) had also been decreased (for example, an ALS patient would be disqualified if they had been diagnosed longer than 2 years ago).
    This leaves a lot of us totally out of treatment or hope.
    Plus, after all this work getting into a trial, you may just get a placebo and watch others who got the “real drug” get better while you are getting sicker. But we would take that chance.

    Now we constantly hear that we are getting closer to cure. But they are still years away as the very slow Clinical Trials that lead to the holy grail of FDA approval move at a snail’s pace.

    “The Right to Try Law” is actually a sham law–Pharmaceutical companies don’t have to let you try any drug in any clinical trial EVEN if it has shown great promise at reversing ALS symptoms (NurOwn), even if every single step required to get permission to try it is followed.
    Since Medicare won’t cover the costs of any drug that has not received formal FDA approval, even if you were lucky enough to be granted the chance to try the drug, the pharmaceutical company charges you whatever amount they want!
    So in the one successful case of RIGHT TO TRY that I have heard of, that one ALS patient had to pay $300,000 to get the drug.
    He now posts videos of himself running and working out in a gym because he got the drug (NurOwn).
    Meanwhile the rest of us are dying–everyday. We cannot wait 6- 12 years for a promising drug to get FDA approval.
    That is why we are advocating for compassionate use and access to the top three most promising drugs still in clinical trials. We will take an chance to continue living. The FDA did release drugs before approval to help with the HIV crisis in the 80’s and saved millions of lives. They can do it again.

  6. Shirley Panebianco says:

    My son was diagnosed in 2013 – December of this year will be 6 years. He was given 2 to 5. If there is anyone who can offer anything for him to try – please IM me. He would be eligible to try anything under the Trump law for those with terminal illness. He was in a trial for Nurown in the early stages, but he did not find it to help in any way and felt it may have made it worse. However, what works for one doesn’t work for all so I’m happy for those who have had success with it.

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