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Trial of new treatment for rare neurological disorder

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6th November, 2023

NSW Health Pathology senior researcher Professor David Brown has received a $2.9 million grant to evaluate immunotherapy treatments for CIDP, a neurological disorder that attacks the body’s nerves.

Cel­e­brat­ed Aus­tralian swim­ming leg­end Michael Klim is one of the country’s most high-pro­file suf­fer­ers of chron­ic inflam­ma­to­ry demyeli­nat­ing polyneu­ropa­thy, or CIDP, describ­ing the debil­i­tat­ing effects of mus­cle wastage in his legs, sen­so­ry loss in his feet and the strug­gle to walk or even stand for long periods.

Klim has recent­ly become a spokesman for a Red Cross Lifeblood cam­paign pro­mot­ing plas­ma dona­tion – a prod­uct used to help treat his con­di­tion and keep him walking.

But the plas­ma treat­ment that pro­vides relief to CIDP suf­fer­ers is expen­sive – cost­ing around $150,000 per patient – and that’s some­thing Pro­fes­sor David Brown, Direc­tor of NSW Health Pathology’s Insti­tute of Clin­i­cal Pathol­o­gy and Med­ical Research (ICPMR) in West­mead, is aim­ing to address.

The Med­ical Research Future Fund (MRFF) has grant­ed Pro­fes­sor Brown $2.9 mil­lion to eval­u­ate immunother­a­py treat­ments for CIDP.

“Peo­ple with chron­ic inflam­ma­to­ry demyeli­nat­ing polyneu­ropa­thy have the insu­la­tion of their nerves stripped off by their dis­ease and this caus­es changes in sen­sa­tion and weak­ness of mus­cles,” Pro­fes­sor Brown explained.

“These patients are treat­ed with intra­venous immunoglob­u­lin, which is very expen­sive and requires life-long treat­ment, often every three weeks.”

Prof Brown said tar­get­ing and elim­i­nat­ing immune cells has sig­nif­i­cant­ly improved out­comes for patients with sev­er­al neu­ro­log­i­cal and inflam­ma­to­ry demyeli­nat­ing diseases.

“This study will deter­mine if remov­ing B‑lymphocytes, a type of white blood cell which makes anti­bod­ies, might be a suit­able ear­ly treat­ment for CIDP and lead to respons­es that will allow less immuno­glo­bin to be used to man­age the con­di­tion. Aside from anec­do­tal evi­dence, there has not been a tri­al to work out whether this approach works in CIDP,” Prof Brown said.

He said there would be ben­e­fits for both patients and the health sys­tem if the ther­a­peu­tic anti­body treat­ment proves to be effective.

“It would mean that instead of hav­ing intra­venous immunoglob­u­lin treat­ment by either week­ly injec­tions or infu­sions every few weeks, treat­ment may be able to be giv­en twice a year,” he said.

“Patients would get back up to 15 days a year of time that they cur­rent­ly ded­i­cate to treat­ment. This approach would also see sig­nif­i­cant cost sav­ings for the health system.”

He says the tri­al is a great exam­ple of how col­lab­o­ra­tion between doc­tors and researchers can work.

“Dif­fer­ent arms of the health sys­tem can work hand in glove with researchers to gen­er­ate and trans­late find­ings in the bed­side to bench and back to bed­side research mod­el that NSW Health Pathol­o­gy plays a cen­tral role in.”

Pro­fes­sor Brown says the research project is now in the final stages of approvals and he expects to be ready to start recruit­ing peo­ple to take part in the treat­ment tri­al in ear­ly 2024.


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