Navigation Links
Candidate drug provides benefit in SMA animal models
Date:6/4/2013

In a new publication that appears in Human Molecular Genetics, the laboratory of Christine DiDonato, PhD reports on their pharmacological characterization of the drug RG3039, demonstrating that it can extend survival and improve function in two spinal muscular atrophy (SMA) mouse models. They have determined the minimum effective dose and drug action, thus contributing to dose selection and exposure estimates for the first studies with RG3039 in humans. As in cellular assays, the animal studies have shown that drug treatment leads to improvement in nuclear gem/Cajal body numbers in motor neurons. Gem loss is a cellular hallmark of fibroblasts derived from SMA patients; gem numbers inversely correlate with SMA disease severity. In addition, the laboratory has shown improved functional outcomes, including treadmill walking and gait dynamics, in animals receiving the drug. The laboratory has been testing RG3039 in SMA mouse models with disease phenotypes ranging from mild to severe.

The collective results suggest that RG3039 positively modifies motor unit pathologies and dysfunction, and that it may have therapeutic benefit for SMA.

Spinal muscular atrophy (SMA) is a devastating hereditary disease that is a leading cause of infant and early childhood mortality. Motor neurons in the spinal cord are affected, resulting in muscle weakness and in many cases breathing problems. SMA is caused by insufficient levels of the survival motor neuron (SMN) protein; depending on the level of SMN protein, patients may have mild to severe forms of SMA. There is currently no cure for the disease.

The goal of early SMA drug discovery programs has been to identify small molecules that induce the SMN gene to produce sufficient levels of protein to improve motor neuron functioning in affected patients. A promising drug candidate is RG3039, which has been shown to increase nuclear gem/Cajal body numbers in cellular assays.

Says DiDonato: "We are very happy that our research was able to assist in the pharmacological characterization of RG3039 and contribute to dose selection and exposure estimates for the first studies with RG3039 in human subjects. We are indebted to my colleague, Nancy Kuntz, MD, and co-author laboratories of Drs. Jasbir Singh and Mike Kiledjian along with Repligen and Families of Spinal Muscular Atrophy (FSMA), who initiated the development of this drug candidate. The depth of our manuscript would not have been possible without this group collaboration. Also, it is reassuring that our results of improved motor unit dysfunction in SMA mice treated with RG3039 mirror the independent findings by the laboratories of Drs. Sumner and Ko. Improved neuromuscular function is an obvious goal for SMA therapies. Thus, this drug candidate warrants further investigation. Overall, this SMA candidate drug is an exciting time as we take another step forward towards developing therapies for SMA patients."


'/>"/>

Contact: Peggy Murphy
pemurphy@luriechildrens.org
773-755-7485
Children's Memorial Hospital
Source:Eurekalert

Related medicine news :

1. Novel drug candidates offer new route to controlling inflammation
2. Presidential candidates should address childhood obesity and bullying, poll says
3. Parc at Piedmont - East Cobb to Host Candidate Forum for Primary Elections
4. New candidate drug stops cancer cells, regenerates nerve cells
5. BWH researchers discover new vaccine candidate for Pseudomonas aeruginosa
6. No Truth to Candidates Claim That Rape Hinders Pregnancy, Experts Say
7. New oral drug candidate for African sleeping sickness
8. Katon Direct, a Healthcare Micro-Marketing Solutions Company, Launches Their New Website, Innovating the way Healthcare Companies Find the Most Qualified Candidates
9. NIH-developed candidate dengue vaccine shows promise in early-stage trial
10. NIH-funded researchers begin trial of Shigella vaccine candidates
11. Drugs targeting blood vessels may be candidates for treating Alzheimers
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:8/17/2017)... ... ... Media, the industry leader in digital marketing for medical practices, has been recognized as one ... the Inc. 5000 for the second time in two years. Shawn Miele, Chief Executive Officer ... for the second year in a row. It’s extremely difficult to make this list once. ...
(Date:8/17/2017)... Yorba Linda, Ca (PRWEB) , ... August 17, 2017 , ... ... by the FDA to the biopharmaceutical industry in 2016 cited deficiencies in data integrity. ... their 21 CFR Part 11 ruling. , Presented as part of the ...
(Date:8/16/2017)... Kalamazoo, Michigan (PRWEB) , ... August 16, 2017 , ... ... Applications are now being accepted. , Students who successfully complete the seven-week long ... a Certified Nurse Assistant (CNA). The average starting salary for a CNA in Kalamazoo ...
(Date:8/16/2017)... (PRWEB) , ... August 16, 2017 , ... Greenfield Advisors ... companies in the U.S. for the second year in a row. The Inc. 5000 ... past three years. , “To be on the list once is a great accomplishment, ...
(Date:8/16/2017)... , ... August 16, 2017 , ... Any Lab ... 5000. The company ranked #4429 on the newly released, 36th annual Inc. 5000 ... a unique look at the most successful companies within the American economy’s most dynamic ...
Breaking Medicine News(10 mins):
(Date:8/7/2017)... 2017 Diplomat Pharmacy, Inc. (NYSE: DPLO ... as president, effective Aug. 7, 2017. ... pursue other interests and will serve as president emeritus during ... served us in multiple leadership roles since he joined Diplomat ... and has provided decisive, strategic leadership which continues to benefit ...
(Date:8/4/2017)... CINCINNATI , Aug. 3, 2017  Agragen, ... science company active in the biopharmaceutical, nutraceutical, and ... its development of its lead drug candidates, AGR131.  ... a proinflammatory cytokine from the blood of patients ... psoriasis, and inflammatory bowel disease. ...
(Date:8/2/2017)... 2017  Life Flight Network and PeaceHealth Oregon Network announced ... care and operational efficiency for patients at hospitals in ... Cottage Grove , and Florence, Oregon ... Life Flight Network work collaboratively to move patients who require ... a time sensitive emergency exists. ...
Breaking Medicine Technology: