Potential ALS Therapy Based on Cancer Treatment Rituximab Shows Promise

Potential ALS Therapy Based on Cancer Treatment Rituximab Shows Promise

Researchers at Ben-Gurion University (BGU) believe that an existing FDA-approved anti-cancer drug, rituximab, could be modified and used to treat amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.

In lab studies with mice, the therapy restored resident immune cells of the central nervous system, which could indicate the potential to increase the life expectancy of ALS patients.

Scientists have not yet found the exact cause of ALS, and experts have focused on developing therapies to reduce symptoms and disease progression. The goal of these therapies is to extend post-onset life expectancy.

Only two drugs have been approved as ALS treatments. Rilutek (riluzole) was approved in 1995 and has helped extend life expectancy by only three to six months.

Twenty-two years later, the FDA approved Radicava (edaravone), which delays disease progression at early stages. Despite these therapeutic advances, more efficient treatments are needed.

Rachel Lichtenstein, PhD, and a team from BGU’s Avram and Stella Goldstein-Goren Department of Biotechnology Engineering have designed a new compound based on the structure of FDA-approved rituximab, whose brand name MabThera is used in treating chronic lymphocytic leukemia and non-Hodgkin’s lymphoma. More recently, its efficacy against autoimmune diseases such as rheumatoid arthritis was discovered.

Rituximab is a monoclonal antibody that was developed to deplete immune B cells in circulation. Taking advantage of its immunomodulatory effect, Lichtenstein hopes to reduce negative immune responses that lead to neuronal death in ALS. This new therapeutic approach could aid the nervous system by improving its tasks of surveillance and removal of dead cells and wastes that can be harmful.

“We found a way to thwart the glial cells from attacking and killing healthy brain cells,” Lichtenstein said in a news release. “Our experimental results on ALS transgenic mice showed a significant increase in life expectancy,” she added.

Because this investigational drug is based on an already approved drug with proven safety, the research team believes it will require fewer preclinical studies to reach the clinical development stage. This could significantly reduce the time it takes to develop the therapy. The researchers are now seeking a pharmaceutical company to partner with to proceed with further studies.

“Our new drug candidate may prove effective in boosting the self-cleansing mechanism of the human brain, thereby improving the lives of millions of people,” said Ora Horovitz, PhD, senior vice president of business development at BGN Technologies. “This could also have major implications on the life expectancy of other neurodegenerative disease patients with Alzheimer’s and Parkinson’s,” she added.

6 comments

  1. Carol Mongiello says:

    Great news. Or is it? How many years will this drug take until it reaches FDA approval? Recent ALS news about Radicava that delays disease progression at early stages. So who makes this distinction of early stages? Is it someone who is affected in only one limb but was diagnosed with AlS 2 years ago? Could disqualify them from some clinical trials. Is it measured by the muscle movement a person has? What about people like my son in just 1 1/2 years he has become totally dependent in all activities of daily living. What about medication for him and probably thousands like him? Give everyone a chance.

      • Alice Melao says:

        Hello Tim,
        This new drug is not FDA approved yet, it is just chemically based on a FDA-approved cancer drug. Many research work is still requiered to confirm is clinical beneficial effects for ALS and to pursue a FDA marketing approval.

    • Alice Melao says:

      Hello Carol,
      I am sorry to ear about your son’s quick disease development.
      Although I am not a doctor and I am not completely aware of the treatment guidelines I believe that all decisions are going to be made by each patient’s clinical team, and most probably their decisions are going to take into account the clinical trials results. For you to have better ideia of the process you should consult your son’s doctor. Hope the best for both of you.

  2. M Bond says:

    I have been diagnosed with ALS going on 18 months,my spirits are great,however my body not so good. Anyway I have monitored this Website for a year and many positive news stories about possible new or retread drugs. The “Big” but is it may not get to me in time to help,why, follow the money. Everything increases the price clinical studies x4,FDA approval , manufacturing,distribution,marketing.
    With approximately 16,000 currently ALS patients in the US, not a money maker for Pharma. All you with ALS know this, you are smart and know the issues.
    Let’s consider shortening trials, fast tract FDA, use network compounding pharmacies to distribute drugs that show promise and consider it a generic, no branding necessary. Walmart dosnt need this inventoried.

    • Carol Mongiello says:

      Is this Mr James Bond? [ I hope you get a laugh ]. My heart goes out to you and your family. My son was diagnosed in 10/15. I have been to Washington DC last summer promoting the Right to Try Act. This act would give people with terminal illnesses the ability to have access to medications that are out there but may not be ready for the “market” yet. I have no idea what ever happened to this act. It sucks to be us. Waiting, waiting for help. I feel so helpless for my son and try to be encouraging to him. One day I thought how many senses do I have. I can see, I can smell, I can hear, I can taste, and I can feel. My son may not be able to move his extremities but he has all the same senses I have and they are all functional and this for today and tomorrow I will feel blessed. Well wishes to you.

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