This Dedicated Scientist Left a Legacy to Fight Cystinosis

According to a recent article in the Sunderland Vibe, the MRC Developmental Pathway Funding Scheme (MRC) was created to carry out the work of Professor Roz Anderson, who recently died of cancer.

Professor Anderson was dedicated to securing a grant from the Medical Research Council to aid in creating and bringing drugs to pretrials that would provide a better quality of life for patients with inherited cystinosis.

About Cystinosis

Cystinosis causes the buildup of cystine in the cells. Amino acids are the building blocks of proteins.

When there is an accumulation of cystine it causes crystals to form that can damage cells. Various organs are affected but the muscles, kidneys, and eyes are most impacted.

The disease is broken down into three distinct types which are nephropathic, intermediate and ocular cystinosis. Each type is caused by mutations in the CTNS gene that send out instructions to create the protein cystinosin.

The other characteristic of the three types of cystinosis is that they are inherited in an autosomal recessive pattern. A person who has an autosomal condition has inherited one mutated gene from each parent. The health of the parent/carrier is generally not affected. One parent carries the recessive (mutated) gene and the other parent carries the dominant gene (normal gene). There is no cure.

Additional information about cystinosis is available here.

How is it Treated?

The treatment used most frequently for cystinosis patients is a bitartrate called cysteamine that reacts with cystine to prevent accumulation of cystine crystals. The drug reduces cystine in white blood cells and muscle tissue.

Additional information about cysteamine is available here.

 A New and Effective Treatment

The novel drug designed by Professor Anderson and her team at the University of Sunderland directs the treatment to the appropriate cells.

In this way the drug improves absorption, limits metabolism, and reduces unpleasant side effects.

 Perpetuating the Legacy

The Head of School, Pharmacy and Pharmaceutical Sciences at the University, Dr. Adrian Moore, commented that the MRC is moving Dr. Anderson’s drug from the University lab to final manufacturing. They hope to have the pre-clinical trials completed within two years. At that time they would begin preparation for clinical trials.

Dr. Moore recalled how hard Dr. Anderson worked to acquire the grant. It is the highest graded grant awarded in recent years by MRC. He said the legacy represents a decade and a half of dedicated work by Dr. Anderson.

The project will be led by Professor Herbie Newell, a world leader in cancer research. Professor Newell is well known for his expertise in taking drugs from discovery to market.

Dr. Newell praised Professor Anderson whose outstanding work in the field of medicinal chemistry will bring hope to many cystinosis patients.

 

 


Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia four years ago. He was treated with a methylating agent While he was being treated with a hypomethylating agent, Rose researched investigational drugs being developed to treat relapsed/refractory AML.

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