After receiving many excellent applications, the winner of the 2024 Dr Simon Newell Early Independent Researcher Award is Dr Maanasa Polubothu for her outstanding accomplishments and remarkable research to improve the lives of children with complex vascular anomalies.
Dr Alex Bonner, Head of Grant Funding at GOSH Charity said:
Dr Maanasa Polubothu’s commitment to improving the lives of children with complex vascular anomalies is evidenced by an abundance of peer-reviewed publications, academic prizes and grants – including a Royal Society research grant and a NIHR GOSH BRC catalyst fellowship early in her career. In 2018, she discovered the genetic cause of arteriovenous malformation (AVM) – a life-threatening vascular tumour in children. This finding had huge translational impact, resulting in the first successful drug treatments for patients with AVM internationally. However, despite progress with treatments, AVM still remains an enormous area of unmet need in the UK. With this award, Dr Polubothu will be able to continue her exploration for new treatments and raise awareness about the condition amongst paediatricians in the UK. Dr Polubothu now leads a national specialist clinic for children with complex vascular anomalies at GOSH, and we are delighted that she has received the 2024 Simon Newell Award, supported by GOSH Charity and RCPCH, in recognition of her impact in this field.
We spoke with Dr Maanasa Polubothu (Consultant in Paediatric Dermatology and Principal Investigator, Genetics and Genomic Medicine at University College London Great Ormond Street Institute of Child Health) about her research, the award and future plans. Maanasa describes the importance of patience when conducting research, the impact of working with patient groups and shares tops tips for aspiring researchers.
What was the motivation behind your clinical research?
Complex vascular anomalies and overgrowth syndromes are truly fascinating conditions cross-cutting both developmental biology and cancer biology. Though a rare disease there is a large cohort of patients in the UK with these incurable and often life-threatening conditions. The discovery of the genetic basis of these conditions as somatic mutations in key oncogenes in recent years has accelerated research efforts and has enabled better understanding of the underlying pathophysiology of disease and importantly enabled new treatments to be developed. This is a very exciting time to be working in this field and I find motivation from both my patients and laboratory research daily! My laboratory research focuses on exploring the processes driving the growth of complex vascular tumours to find new ‘druggable’ targets and ultimately treatments. My clinical research focuses on improving our understanding of the multi-system burden of disease coupled with improving access to genetic testing and access to repurposed cancer therapies for patients in the UK.
What does it mean to you to involve paediatric patients and families in the development and delivery of research?
Patient and public involvement has been fundamental to my research journey. Research funding in rare disease can be challenging and much of my early work was funded with pump-priming grants from patient groups. There are three organisations that I currently work closely with: Butterfly AVM; the UK Lymphangiomatosis; and Gorham’s Disease Alliance (UK-LGDA) and GO-PI3KS. In addition, working with patient groups has greatly informed the direction of my research and shaped my research priorities to align with their goals. This direct feedback has been invaluable, as a researcher you have your research priorities based on your knowledge and experience, but this may differ from the priorities of patients and you can only understand patients' priorities by working with them.
What have been your key takeaways from your research so far?
Patience is really key to conduct well designed research and required in abundance to follow through on projects which may not go as you had initially anticipated. It is important to be flexible and continually adapt and evolve in the face of challenges or novel sometimes unanticipated data. The dynamic and unpredictable nature of science is really what makes it truly rewarding.
What does it mean to you to have won the Simon Newell award?
It is such an unexpected privilege to receive this award. I am honoured to be listed alongside the many brilliant former recipients and Dr Simon Newell himself, who have all driven forward a wealth of groundbreaking research across many diverse disciplines in child health. I am especially grateful to be recognised whilst working in rare disease and I hope this award will raise awareness about the challenging conditions I treat.
What motivated you to get into Paediatric Research?
I have always been very inquisitive which lends well to both clinical and laboratory research. I really enjoyed laboratory work during my intercalated BSc and MSc but it wasn’t until I started my PhD that I truly understood the joy of the bedside to bench and back to bedside loop. I relished being able to see patients in clinic one day, ponder research questions and then to directly address these in the lab the following day and if lucky to relay important results to patients and directly influence their ongoing management.
What are your plans for your research career?
I was recently awarded research grants from the Royal Society of Medicine, the MRC and a NIHR GOSH BRC fellowship to continue my work on unpicking the mechanisms by which oncogenic mutations in early development drive the evolution of vascular anomalies. We will be working with cellular and animal models. and testing new treatments in vitro and in vivo. In addition, I will be working to streamline patient pathways and ensure early genetic testing and access to targeted therapies for patients with complex vascular anomalies and overgrowth syndromes in the UK.
What advice would you give to aspiring researchers?
The life of a clinical academic is largely filled with obstacles and rejection with the occasional seemingly serendipitous success. Maintaining motivation whilst balancing both a busy clinical schedule and research can be very taxing and in times of rejection it can be difficult to see a path forward. In such times, the best advice I can give would to be patient and kind to yourself; regroup, try to draw learning points, speak to your mentors and let your patients and unanswered research questions be your driving motivation.
Secondly, I would suggest before jumping in, take your time to find a project, supervision team and environment you feel you can flourish in. I was extremely fortunate to find a wonderful PhD supervisor early in my career who was not only a huge inspiration in that she is a truly brilliant clinician and scientist with an infectious enthusiasm for science, but, who also generously invested time and care in me personally. She is a true advocate for early career researchers and women in science and without her belief in me I wouldn’t have believed I could pursue clinical academia. Most senior academics are happy to be approached and offer advice and mentorship; don’t be afraid to approach several people whilst trying to find the right project and place for you.