Another step toward sickle cell disease treatment

July 10, 2014 by Eric Peters, Virginia Commonwealth University
Martin Safo, Ph.D., and Richmond Danso-Danquah, Ph.D.

(Medical Xpress)—A compound discovered at Virginia Commonwealth University has taken yet another step closer to becoming the only approved drug in the world that is therapeutically effective in managing adult sickle cell disease by targeting hemoglobin.

Named Aes-103, the compound is the focus of the lead program at AesRx, a biopharmaceutical company dedicated to the development of novel drugs. It was announced this week that AesRx was purchased by Baxter International, a global health care company with expertise in medical devices, pharmaceuticals and biotechnology.

"This is so important that I cannot underestimate its value," said Donald Abraham, Ph.D., Alfred and Frances P. Burger Emeritus Professor of Medicinal Chemistry and Biological Chemistry at VCU, who is an original developer of the compound. "Without this financial support, the advanced would have taken many years longer."

The compound, originally patented by VCU under the name 5-HMF, was developed by a team from the VCU Institute for Structural Biology and Drug Discovery, an interdisciplinary research center spanning the School of Medicine and the School of Pharmacy. The team included Martin Safo, Ph.D., associate professor of ; Richmond Danso-Danquah, Ph.D., assistant professor of medicinal chemistry; and Abraham, emeritus director of the institute.

Relabeled as Aes-103, the compound was licensed by VCU Innovation Gateway to AesRx - a startup company at the time - which began clinical trials.

"We are very grateful for the role VCU has played in the development of Aes-103," said Steve Seiler, AesRx founder and CEO. "As a small company focusing on a big unmet medical need, AesRx needed to be pragmatic in its development strategy for Aes-103 and adjust as new data and financing strategies emerged. VCU worked collaboratively with us on this effort and was a true partner with AesRx all along the way."

VCU's liason to the company has been VCU Innovation Gateway, a university resource that facilitates commercialization of university inventions and supports university research through collaborative agreements.

"Without Innovation Gateway at VCU, and the efforts of our former president Dr. Eugene Trani and his establishment of the Biotechnology Research Park, this invention would have never reached clinical trials," Abraham said. "I am very grateful to have had the opportunity for our basic research to be translated into a potentially useful compound for the treatment of ."

The Aes-103 program is currently in a Phase 2 clinical trial as part of an ongoing collaboration with the NIH's National Center for Advancing Translational Sciences (NCATS) through its Therapeutics for Rare and Neglected Diseases (TRND) program.

Sickle cell disease is a recessive disorder of the hemoglobin that can lead to a wide range of serious, sometimes life-threatening conditions, including chronic hemolytic anemia, chronic pain and acute painful crisis, stroke, acute chest syndrome and cumulative damage to tissues and organs. More than 100,000 people in the United States are afflicted with .

Aes-103 is a first-in-class, oral, small molecule compound. Early studies indicate the compound may work by binding to and stabilizing the relaxed state of hemoglobin and increasing oxygen affinity and stabilization, thereby reducing the sickling of red blood cells which, in turn, may reduce sickling-related outcomes such as vaso-occlusive crisis, pain, severe anemia and fatigue.

Explore further: Research leads to possible new treatment for sickle cell disease

Related Stories

Research leads to possible new treatment for sickle cell disease

September 23, 2013
There's a new ray of hope for those battling sickle cell disease thanks to a compound that was discovered at Virginia Commonwealth University.

First drug candidate from NIH program acquired by biopharmaceutical company

July 9, 2014
A drug candidate developed by researchers at the NIH's National Center for Advancing Translational Sciences (NCATS) and its collaborators to treat sickle cell disease has been acquired by Baxter International's BioScience ...

New compound discovered that rapidly kills liver cancer

March 14, 2012
Scientists have identified a new compound that rapidly kills hepatocellular carcinoma (HCC) cells, the most common form of liver cancer and fifth most common cancer worldwide, while sparing healthy tissue. The compound, Factor ...

Novel therapy helps ease pain and suffering for sickle cell patients

September 25, 2012
Chronic, debilitating pain and potential organ failure are what approximately 100,000 sickle cell patients in the United States live with each day. Yutaka Niihara, M.D., M.P.H. - lead investigator at The Los Angeles Biomedical ...

Researchers reveal potential treatment for sickle cell disease

November 2, 2011
A University of Michigan Health System laboratory study reveals a key trigger for producing normal red blood cells that could lead to a new treatment for those with sickle cell disease.

Recommended for you

More surprises about blood development—and a possible lead for making lymphocytes

January 22, 2018
Hematopoietic stem cells (HSCs) have long been regarded as the granddaddy of all blood cells. After we are born, these multipotent cells give rise to all our cell lineages: lymphoid, myeloid and erythroid cells. Hematologists ...

How metal scaffolds enhance the bone healing process

January 22, 2018
A new study shows how mechanically optimized constructs known as titanium-mesh scaffolds can optimize bone regeneration. The induction of bone regeneration is of importance when treating large bone defects. As demonstrated ...

Bioengineered soft microfibers improve T-cell production

January 18, 2018
T cells play a key role in the body's immune response against pathogens. As a new class of therapeutic approaches, T cells are being harnessed to fight cancer, promising more precise, longer-lasting mitigation than traditional, ...

Weight flux alters molecular profile, study finds

January 17, 2018
The human body undergoes dramatic changes during even short periods of weight gain and loss, according to a study led by researchers at the Stanford University School of Medicine.

Secrets of longevity protein revealed in new study

January 17, 2018
Named after the Greek goddess who spun the thread of life, Klotho proteins play an important role in the regulation of longevity and metabolism. In a recent Yale-led study, researchers revealed the three-dimensional structure ...

The HLF gene protects blood stem cells by maintaining them in a resting state

January 17, 2018
The HLF gene is necessary for maintaining blood stem cells in a resting state, which is crucial for ensuring normal blood production. This has been shown by a new research study from Lund University in Sweden published in ...


Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.