Navigation Links
HSCI researchers identify another potential ALS treatment avenue

Cambridge, MA, Aug 6 - A series of studies begun by Harvard Stem Cell Institute (HSCI) scientists eight years ago has lead to a report published today that may be a major step forward in the quest to develop real treatments for amyotrophic lateral sclerosis, ALS, or Lou Gehrig's disease.

The findings by Harvard professor of Stem Cell and Regenerative Biology (HSCRB) Kevin Eggan and colleagues also has produced functionally identical results in human motor neurons in a laboratory dish and in a mouse model of the disease, demonstrating that the modeling of human disease with customized stem cells in the laboratory could someday relatively soon eliminate some of the need for animal testing.

The new study, published today in Science Translational Medicine, suggest that compounds already in clinical trials for other purposes may be promising candidate therapeutics for ALS. The Harvard authors found that genetically intervening in the pathway these drugs act on increased survival time of an ALS animal model 5-10 percent, and while that is a long way from curing the universally fatal neurodegenerative disease, "any ALS patient would be excited about this extended life span," said Eggan, who pioneered the disease in a dish concept.

Sophie De Boer, a graduate student in Eggan's lab, is the first author on the Science Translational Medicine paper.

This latest finding is expected to push towards clinical studies the second major ALS discovery from Eggan's lab in less than a year. The HSCI stem cell biologist, and his neuroscience and neurology collaborators at Massachusetts General Hospital and Boston Children's Hospital, are preparing for a phase I clinical trial of a medication already approved for epilepsy which Eggan and colleagues discovered quiets disease related electrical excitability in the motor neurons effected in ALS.

In a paper in 2007, Eggan and colleagues demonstrated that glial cells, background cells in the nervous system, were involved in motor neuron degeneration in a mouse model of ALS. And the following year the researchers reported that the same thing was happening in human motor neurons made from patient stem cells, and proposed that prostanoid molecules, a group of substances involved in inflammation in everything from pain to pregnancy, might be playing a role in the glial cells.

Today the researchers reported they have confirmed there is a change in prostanoid receptors in the gial cells playing a role in ALS, and with genetic and chemical experiments they showed that this is playing a role in ALS. They further report that when the effected receptor is blocked, the ALS damage done by the glial cells is reduced.

This latest work, says Eggan, first done in human motor neurons in a dish, and then in a mouse model of ALS, "says that indeed this stem cell model was predictive of something that can happen inside a whole animal, and its important because it demonstrates that this is really an important target for an ALS therapeutic. If we can inhibit this receptor in an ALS patient, we might slow down the progression of the disease, and that would be a huge step."

Eggan said "one feature of the glial cells in ALS that attack motor neurons is that they have higher expression of this prostanoid receptor. Removing just one of the two copies of the receptor in the glial cells had an effect on extending the life span" of the ALS mice," Eggan said, and "inhibition by a drug is unlikely to have an effect as complete as a knockout in the mice."

Eggan said that experiments on human stem cell-generated ALS motor neurons also show that "if we inhibit that receptor in the ALS cells with a chemical, those cells lose their toxicity to motor neurons

"This is a very exciting period for those whose lives are threatened by ALS, and it is exciting for my lab," Eggan said. "First we recently identified a pathway that we think is important for degeneration inside the motor neuron, and now we've found this pathway in cells outside the motor neuron. This has potential to have a very substantial effect on what's happening in ALS."

The research was funded by Project ALS, the New York Stem Cell Foundation, P2ALS, and the Howard Hughes Medical Institute.


Contact: B. D. Colen
Harvard University

Related biology news :

1. Study by UC Santa Barbara researchers suggests that bacteria communicate by touch
2. UC Santa Barbara researchers discover genetic link between visual pathways of hydras and humans
3. Researchers attempt to solve problems of antibiotic resistance and bee deaths in one
4. UNH researchers find African farmers need better climate change data to improve farming practices
5. Ottawa researchers to lead world-first clinical trial of stem cell therapy for septic shock
6. Researchers uncover molecular pathway through which common yeast becomes fungal pathogen
7. Researchers print live cells with a standard inkjet printer
8. Columbia Engineering and Penn researchers increase speed of single-molecule measurements
9. Researchers reveal how a single gene mutation leads to uncontrolled obesity
10. Researchers discover novel therapy for Crohns disease
11. New paper by Notre Dame researchers describes method for cleaning up nuclear waste
Post Your Comments:
Related Image:
HSCI researchers identify another potential ALS treatment avenue
(Date:11/9/2015)... 09, 2015 ... the "Global Law Enforcement Biometrics Market ... --> ) has announced the ... Biometrics Market 2015-2019" report to their ... ( ) has announced the addition ...
(Date:10/29/2015)... , Oct. 29, 2015   MedNet Solutions ... the entire spectrum of clinical research, is pleased to ... High Tech Association (MHTA) as one of only three ... the "Software – Small and Growing" category. The Tekne Awards ... who have shown superior technology innovation and leadership. ...
(Date:10/29/2015)... , Oct. 29, 2015  Connected health pioneer, ... the explosion of technology-enabled health and wellness, and the ... book, The Internet of Healthy Things ... or smartphones even existed, Dr. Kvedar, vice president, Connected ... health care delivery, moving care from the hospital or ...
Breaking Biology News(10 mins):
(Date:12/1/2015)... ... December 01, 2015 , ... Park Systems ... an add-on scanning ion conductance microscopy module to Park NX10 that is the ... , Park SICM benefits virtually all materials characterization that require measurements in liquid ...
(Date:12/1/2015)... SAN DIEGO , December 1, 2015 Dr. Harry Lander ... serving as Chief Science Officer and recruits ... Dr. Harry Lander , President of Regen, expands his role ... and recruits five distinguished scientists to ... of Regen, expands his role to include ...
(Date:11/30/2015)... ... November 30, 2015 , ... Global Stem Cells Group ... of a new closed system for isolating adipose-derived stem cells. The announcement starts a ... of adipose tissue. SVF is a component of the lipoaspirate obtained from liposuction of ...
(Date:11/30/2015)... 2015 Partnership includes an MPP ... for the u niversity , s ... treatment s cale - up through ... Africa , where licensees based anywhere in the world will have ... --> Africa , where licensees based anywhere in the world ...
Breaking Biology Technology: