NHS patients in England with von Hippel-Lindau disease – a rare genetic disease that causes tumours in major organs, including the brain and spine – can now benefit from the first-ever therapy which could shrink their tumours to help them avoid high-risk surgery.
In clinical trials of belzutifan, 95 per cent of patients did not experience any growth in their tumours in two years of taking the treatment, and 56 per cent of patients’
On average, people with von Hippel-Lindau disease do not live past their early 60s, with tumours in the brain and spinal cord being the leading cause of death.
Belzutifan, which is manufactured by MSD UK, is taken as a tablet three times a day and targets and controls three different types of tumours at the same time.
The genetic condition causes an abundance in proteins that can starve cells of oxygen, which leads normal cells to transform into tumour cells. Belzutifan stops this protein from starving cells of oxygen and prevents the growth of tumours.
Research into the genetic disease and this oxygen-starving protein in cancer development won the Nobel Prize for Physiology and Medicine in 2019, with belzutifan becoming the first available treatment that inhibits the protein from starving cells of oxygen.
Rachel Amner, 49, from South East London, is one of nine in her family with VHL, across four generations.
She said: “My grandfather was blinded by VHL tumours before dying from a brain tumour. Between myself and four family members with VHL, we’ve had over 20 major surgeries, including 12 brain surgeries, three spinal surgeries, the removal of a kidney, pancreas and three eyes removed – my cousin lost both and is now totally blind.
“Living with VHL means that our family lives with the anxiety of not knowing where the next life-changing blow is coming from. Every single major lifestyle adjustment we have made due to invasive VHL surgery, from blindness to poor mobility, is a challenge we live with every day.
“Belzutifan gives us the reassurance of stabilising our conditions without crushing our life hopes with further tumour diagnoses.”
Chair of VHL UK/Ireland, Graham Lovitt, said: “We are delighted by NICE’s recommendation of belzutifan (Welireg) under the managed access agreement, a significant milestone in the fight against this rare disease.
“This decision brings hope to patients and their families, offering them an alternative treatment option, where others become too high risk. VHL UK/Ireland remains committed to supporting those affected by the condition and helping everyone who can benefit from this breakthrough medicine to access it.”
Benson Fayehun, Head of Oncology at MSD UK, added: “We are delighted that eligible patients in England and Wales will be able to access belzutifan. VHL disease is a complex and devastating genetic condition, with patients often acting as carers for their family members, who may also have a VHL diagnosis.
“Until now, these patients have had few treatment options, typically limited to invasive procedures. This decision provides a welcome option for patients, reducing the need for and impact of numerous surgeries.”
