NHS Introduces ‘Life-Changing’ Sickle Cell Treatment for Thousands
Thousands of sickle cell disease patients in England can now access a new treatment, Voxelotor, on the NHS. Approved by NICE (National Institute for Health and Care Excellence), this daily tablet could benefit up to 4,000 patients by reducing the need for blood transfusions and hospital visits. This is particularly useful for those with severe reactions to transfusions or who prefer to avoid them.
Sickle cell disease affects around 17,000 people in England, mainly those of Black African, Caribbean, Middle Eastern, and South Asian heritage. The condition causes red blood cells to become sickle-shaped, leading to haemolytic anaemia, severe pain, organ damage, strokes, and sight loss.
NHS chief executive Amanda Pritchard highlighted the treatment as a major breakthrough, set to improve lives and reduce hospital dependency. Approved for individuals aged 12 and older, Voxelotor can be used alone or with hydroxycarbamide. Clinical trials showed 51% of patients had improved haemoglobin levels, and 74% saw overall health improvements.
NHS England’s Professor Bola Owolabi and NICE's Helen Knight praised the drug’s potential to reduce health disparities. Dr. Jennifer O’Connor, a trial participant, reported significant improvements in energy, pain levels, and quality of life since starting Voxelotor.
Scans Show 'Wonder' Treatment Shrinks Woman's Brain Tumour
A revolutionary new treatment has shown remarkable results in a woman with a brain tumour, with nearly complete regression in less than a week. The 57-year-old experienced dramatic improvement after undergoing CAR-T therapy, a treatment that enhances the body's immune response.
She was one of three glioblastoma patients in a study from March to July 2023, all of whom saw significant tumour reduction. Dr. Elizabeth Gerstner from Massachusetts General Hospital noted the dramatic and rapid response in these patients, highlighting the progress but also the need for further research.
Glioblastomas are highly aggressive brain tumours affecting around 3,200 people annually in the UK. Traditional treatments include surgery, radiotherapy, and chemotherapy. This new study, published in the New England Journal of Medicine, evaluated CAR-T therapy's effectiveness in treating this condition. Originally used for certain blood cancers, CAR-T therapy trains the immune system to target cancer cells. The woman experienced "near-complete tumour regression" in just five days. The other patients also saw reductions, with a 72-year-old man's tumour shrinking by over 18% in two days and by 60.7% by day 69, sustaining for over six months.
Dr. Marcela Maus of Massachusetts General Hospital called the results promising, though emphasised that curing the disease remains the ultimate goal.
Individuals with HIV Now Eligible to Donate Sperm and Eggs
Changes to UK laws now allow same-sex couples where one or both partners are HIV-positive with a non-transmissible form of the virus to donate sperm or eggs for parenthood. This update aligns with scientific advancements that confirm the safety of such donations due to highly effective antiretroviral medications, which nearly eliminate the risk of transmission.
This policy shift expands reproductive rights and is part of broader efforts to enhance access to in vitro fertilisation (IVF) services and promote equality. Previously, regulations only permitted HIV-positive men to donate sperm to female partners. The new rules apply to people who have maintained an undetectable viral load for a sustained period and have been on antiretroviral treatment for at least six months. Recipients must be fully informed of the donor's HIV status and give their consent.
Additionally, the government is addressing cost disparities in fertility treatments, particularly for female same-sex couples who undergo the shared motherhood IVF process. These couples have faced higher screening fees than heterosexual couples, a financial burden that will now be removed to foster a more equitable system.
Officials anticipate that these changes will decrease the stigma associated with HIV and underscore the capability of individuals with the virus to lead fulfilling lives. Clinics are expected to begin implementing these new guidelines shortly, with the Human Fertilisation and Embryology Authority set to issue detailed guidance on navigating the updated laws.