Stem cell research tackles Lou Gehrig’s disease

Embracing the concept that the whole is greater than the sum of its individual parts, an $11.5 million “disease team” grant has been awarded to UCSD to fast track stem cell research on Amyotrophic Lateral Sclerosis (ALS) — also known as Lou Gehrig’s disease. The goal is to advance basic research to human clinical trials within four years.

Funding is from the California Institute for Regenerative Medicine (CIRM), the state stem cell agency created by voter passage of Proposition 71. In a public meeting on June 23, local scientists, health advocates and CIRM leadership described the grant’s research focus.

ALS is a progressive, fatal disease in which motor neurons, the nerve cells that control voluntary muscle contractions, simply die. Over time, ALS leads to extreme muscle weakness, paralysis and death.

“Like most cells in the body, motor neurons do not live in isolation; they live in an environment, surrounded by other type of non-neuron cells,” said Larry Goldstein, Ph.D., professor of cellular and molecular medicine at UCSD School of Medicine, Howard Hughes Medical Institute investigator and director of the UCSD Stem Cell Program.

“Therefore, one of the ways you might imagine to eliminate (disease) spread would be by supporting the ‘neighborhood’ rather than replacing the neurons themselves.”

This has led to interest in exploiting a special type of cell called an astrocyte progenitor. Astrocytes are glial cells, a family of cells that support the proper functioning and insulation of neurons.

Astrocytes, in particular, help with neurotransmissions and neuronal metabolism. Previous studies indicate that transplanting healthy glial cells into patients could be a possible treatment for ALS, and animal studies have shown that astrocytes possess particular promise in this regard.

Co-principal investigators on the grant are Goldstein; Martin Marsala, M.D., professor in the UCSD School of Medicine’s department of anesthesiology; and Sam Pfaff, Ph.D., a professor in the Salk Institute’s Gene Expression Laboratory. They will lead researchers in studies of two methods of administering progenitors in animal models and test the safety and efficacy of these approaches, with the goal of providing proof-of-principle and laying the groundwork for clinical trials.

“Any novel treatment option would not only be clinically competitive, but could have a major impact for thousands of patients currently battling this disease,” Don Cleveland, Ph.D., professor of medicine, neurosciences and chair of the UCSD Department of Cellular and Molecular Medicine, stated in a news release. Cleveland is also head of the Laboratory of Cell Biology at the Ludwig Institute for Cancer Research based at UCSD. “This approach has the potential to lead to the development of new therapies that could significantly extend the life span of individuals living with this disease, and improve their quality of life.”

In addition to basic research, animal data and pre-clinical aspects of the studies, another disease-team member is Carlsbad-based Life Technologies Corp., which will provide expertise in stem cell biology, cell separation, next-generation sequencing and scalable bioproduction.

“The goal is to develop a clinically compliant process for preparation of human astrocyte precursors,” said Mark Bonyhadi, Ph.D., director of clinical business development for cell therapy systems at Life Technologies.

Approximately 30,000 people in the United States are affected with ALS, with 5,600 new cases diagnosed each year. The causes of ALS are unknown; there is no cure. Only one drug — Rilutek — is currently approved by the U.S. Food and Drug Administration for treating ALS. Rilutek extends the life span of ALS patients by a maximum of three months.

Related posts:

  1. $11.5 million grant to aid UCSD stem cell researchers tackling Lou Gehrig’s disease
  2. SRI eliminating the controversy in stem cell research
  3. Stem cell guru to discuss book
  4. La Jolla groundbreaking a landmark for stem cell collaboration
  5. Scripps research advances stem cell work

Short URL: http://www.delmartimes.net/?p=7007

Posted by marylajolla on Jul 1, 2010. Filed under Archives. You can follow any responses to this entry through the RSS 2.0. You can leave a response or trackback to this entry

Leave a Reply

Archives

Facebook

Bottom Buttons 1

Bottom Buttons 2

Bottom Buttons 3

Bottom Buttons 4

Bottom Buttons 5

Bottom Buttons 6

LA JOLLA NEWS

RSS LA JOLLA NEWS

  • La Jolla Library welcomes new chief Shaun Briley
    For La Jolla Riford Library’s new head librarian, Shaun Briley, books have been a part of his life and career, in some form or another, every step of the way. […]
  • Tangerine trees, marmalade skies for Beatles-inspired Patrons of the Prado gala in Balboa Park, San Diego
    “Lucy in the Sky with Diamonds” was the theme of the Patrons of the Prado gala on July 12, 2014 in Balboa Park, San Diego. Beatles-inspired music came from Wayne Foster Entertainment. Sandy Redman and Jeanne Jones served as event chairs. 2014’s beneficiaries are the Reuben H. Fleet Science Center, San Diego Museum of Art and The Old Globe Theatre. […]
  • La Jolla’s Best Bets for events July 31- Aug. 7
    Museum of Photographic Arts (MOPA) offers a crash course in starting a photography collection 6 p.m. Thursday Aug. 7. at the Ligne Roset Showroom, 7726 Girard Ave. MOPA assistant curator Chantel Paul and director of jdc Fine Art Jennifer DeCarlo will share professional insights. […]

RANCHO SANTA FE NEWS

RANCHO SANTA FE NEWS

RSS RANCHO SANTA FE NEWS

  • It’s no stretch for Rancho Santa Fe yoga instructor to ‘give back’
    As one of North County’s most prolific yoga instructors, Stacy McCarthy doesn’t just teach yoga, she lives the yoga lifestyle. Selfless service, or seva in Sanskrit, is an important concept of yoga — one that the Rancho Santa Fe resident practices daily. “When we give and we give from the heart, there’s nothing more gratifying than that,” McCarthy said. […]
  • Rancho Santa Fe resident’s Gen 7 wines earn top honors
    Rancho Santa Fe resident Tim Bacino’s Gen 7 Wines is on a hot streak, his varietals winning several awards this summer in California wine competitions. […]
  • Torrey Pines High School baseball alum Taylor Murphy excelling in pro ball
    It was during a breakout senior year at Torrey Pines High that Taylor Murphy first popped up on the radar of professional scouts. Shortly after graduation, Murphy was selected by the San Diego Padres in the 40th round of the June 2011 amateur draft. He declined, honoring a commitment to the University of the Pacific. Three years later, Murphy got another sho […]