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:10/13/2017)... ... 13, 2017 , ... Many families have long-term insurance that covers care for ... a waiver for care if the client has a cognitive impairment diagnosis. , ... care, is often waived, so the benefits from their insurance start immediately,” said Mechell ...
(Date:10/13/2017)... ... October 13, 2017 , ... Talented ... the lowdown on sciatica in a new episode of "Success Files," which is ... events and innovation and investigates each subject in-depth with passion and integrity. , ...
(Date:10/12/2017)... ... October 12, 2017 , ... CitiDent and San Francisco dentists, ... cutting-edge Oventus O2Vent technology. As many as 18 million Americans are estimated to ... breathing. Oral appliances can offer significant relief to about 75 percent of people ...
(Date:10/12/2017)... , ... October 12, 2017 , ... ... a leader in post-acute health care, have expanded their existing home health joint ... Health. , AccentCare has been operating a joint venture home health company with ...
(Date:10/12/2017)... , ... October 12, 2017 , ... On Saturday, October ... treadmill relay – Miles by Moonlight to raise money for the American Heart Association ... more. , Teams will work together to keep their treadmills moving for 5 ...
Breaking Medicine News(10 mins):
(Date:9/22/2017)... -- As the latest Obamacare repeal effort moves is debated, ... and Lindsey Graham (R-SC) medical device market ... is in an odd place.  The industry wants repeal ... on medical device sales passed along with the Affordable ... visits and hospital customers with the funding to invest ...
(Date:9/19/2017)... 2017 HistoSonics, Inc., a venture-backed medical device company developing a non-invasive, robotically assisted, ... three leadership team developments today:   ... ... Tom Tefft ... Veteran medical device executive Josh Stopek , PhD, who has ...
(Date:9/18/2017)... ALLENTOWN , Pa. and KALAMAZOO, Mich. ... Allentown, Penn. , and OptiMed Specialty Pharmacy ... a business partnership to offer a strategic hub service ... PMD Healthcare,s highly sought-after personal spirometer, Spiro PD 2.0, ... A spirometer is a medical device used ...
Breaking Medicine Technology: