Frances Platt facts for kids

Frances Mary Platt is a brilliant biologist. She studies how our bodies work at a tiny level, focusing on chemistry and medicines. Her main work is about rare genetic problems called lysosomal storage diseases. These conditions can cause serious health issues, often affecting the brain over time.

Frances Platt is a Professor at the University of Oxford in the UK. She is also the Head of the Department of Pharmacology there. Her important work has earned her special recognition. She became a Fellow of the Academy of Medical Sciences in 2011. In 2021, she was also made a Fellow of the Royal Society, which is a very old and respected science group.

Early Life and Learning

When Frances Platt was a child, she loved insects! Her father was a doctor.

She went to Imperial College London for her first university degree. There, she studied zoology, which is the study of animals. After that, she continued her studies at the University of Bath. For her PhD, she researched how special proteins called monoclonal antibodies affected the glands of a type of moth called Manduca sexta.

Later, Frances Platt became a postdoctoral researcher. This means she did more research after her PhD. She worked at the Washington University School of Medicine in the US. There, she looked into problems caused by too much of certain fats called glycosphingolipids. During her last year, her boss moved to a company called Monsanto. This gave Frances Platt a chance to learn about research in the drug industry. She also started working on medicines that fight viruses.

Her Amazing Research Career

In 1986, Frances Platt moved to the US. She worked as a postdoctoral researcher at Washington University School of Medicine. She studied molecules on the surface of special cells called B cells.

In 1987, her research leader, Joseph Davie, moved to the drug part of Monsanto. This is where Frances Platt learned a lot about how research works in companies. She also started working on antiviral drugs, which help fight viruses.

In 1989, she came back to the UK. She became a Senior Research Fellow at the University of Oxford. She worked in the Glycobiology Institute, which is part of the Department of Biochemistry. In 1996, she received a special five-year research grant from the Lister Institute of Preventive Medicine.

In 2008, she became a Professor of Biochemistry and Pharmacology. Then, in 2020, she became the Head of the Department of Pharmacology.

Understanding Lysosomal Storage Diseases

Professor Platt's main research is on lysosomal storage diseases. To understand these, think of a cell like a tiny house. Inside this house, there are small "recycling centers" called lysosomes. Lysosomes are supposed to break down large molecules that the cell no longer needs.

But sometimes, these recycling centers don't work properly. When this happens, molecules build up inside the lysosome. This build-up causes lysosomal storage diseases.

Finding New Treatments

Frances Platt is an expert in glycosphingolipids (GSLs). These are fats that can cause problems in lysosomal storage diseases. With her colleagues, Terry Butters and Raymond Dwek, she found a new way to treat these genetic disorders.

Their work led to a medicine called miglustat. This drug is now approved to treat type 1 Gaucher disease and Niemann-Pick disease type C.

She also helped start a company called IntraBio. Her research has continued to help develop new medicines. For example, a drug called Aqneursa was approved in September 2024 by the FDA. This drug helps treat Niemann-Pick disease type C.

Currently, her research focuses on how lysosomal storage diseases develop. She also looks at how to treat them. She is also studying how these diseases might be connected to Parkinson's disease.

Awards and Special Recognitions

Frances Platt has received many awards for her important work:

• 1998: Gaucher Association Alan Gordon award
• 1999: Horst-Bickel Award
• 2011: Elected Fellow of the Academy of Medical Sciences
• 2013: Royal Society Wolfson Merit Award
• 2016: Wellcome Trust Investigator
• 2018: Elected a Member of the Academia Europaea (MAE)
• 2021: Elected Fellow of the Royal Society

Important Research Papers

Here are some of the key research papers Frances Platt has published:

• First paper identifying the drug NB-DNJ (miglustat) as a glycosphingolipid biosynthesis inhibitor - This paper showed that miglustat could stop the body from making too many glycosphingolipids.
• First study to show that miglustat crosses the blood-brain barrier and can reduce glycosphingolipid storage in the brain - This research proved that miglustat could reach the brain and help reduce the build-up of fats there. This was important for treating brain-related conditions.
• Study reporting the pivotal clinical trial of miglustat in patients with type 1 Gaucher disease and demonstrated efficacy. This study led to approval of miglustat by the EMA and FDA in 2002/3 - This paper showed that miglustat worked well for patients with Gaucher disease. This led to the drug being approved for use in Europe and the US.
• A pivotal study showing that activation of the innate immune system is a common hallmark of lysosomal disorders of the brain and a new therapeutic target - This important study found that brain inflammation is a key part of many lysosomal disorders. This discovery opened new ways to treat these diseases.