A new gene therapy is transforming the lives of patients with a debilitating hereditary condition that causes painful and even fatal levels of swelling. The therapy led to significantly promising results in its first trial involving humans, with patients involved in the study reporting “dramatic improvements” in their symptoms that allowed them to stop taking long-term medication and resume their regular day-to-day activities.
Hereditary angioedema is a rare condition that arises from a genetic mutation and causes leaky blood vessels in patients. This leads to random bouts of swelling that can affect the hand, feet, throat, bowels, mouth and lips as often as two times a week and last for up to days. The condition is disfiguring when it affects the face. It can also leave patients bedridden if the swelling occurs in their bowels. In severe cases, swelling of the throat can cause suffocation and eventually death.
Hereditary angioedema occurs when a C1 inhibitor gene mutation stops the gene’s functionality, resulting in kallikrein protein overproduction and causing a protein called bradykinin to build up. This bradykinin buildup is responsible for the leaky blood vessels and swelling that characterizes hereditary angioedema.
The clinical trial at Te Toka Tumai, Auckland City Hospital involved infusing patients diagnosed with this hereditary disorder with nanolipids designed by the gene-editing tool Crispr to invade liver cells and remove the kallikrein gene. Knocking out the gene stopped bradykinin overproduction and buildup, resulting in a significant reduction in symptoms.
A Suffolk resident who took part in the trial noted that the unpredictability, randomness and potential severity of the swelling attacks made it nigh impossible for him to live his life. He revealed that he hadn’t had any swelling bouts for 18 months and reported significant improvement in his mental and physical well-being.
According to New Zealand nurse and clinical trial participant, Judy Knox, the therapy worked “like a magic wand.” The nurse suffered from vomiting, abdominal swelling, and severe pain that often lasted some days before she received a hereditary angioedema diagnosis.
Knox says the experimental therapy has changed her life and allowed her to wean herself off the often-unreliable androgen medication she used to control her symptoms.
Although the trial was small and only involved 10 patients, its findings have opened the door to new and potentially more effective treatments for people suffering from hereditary angioedema. Cambridge University hospital clinical immunology and allergy consultant Dr Padmalal Gurugama notes that the therapy is transforming the lives of patients. One of his patients had swelling attacks every three weeks but hasn’t had any attacks in the 18 months since they received the treatment despite not taking any medications.
The researchers reported their findings in the “New England Journal of Medicine” and are hoping to recruit patients for a phase 3 trial in 2025.
As more research teams at different entities such as Renovaro BioSciences Inc. (NASDAQ: RENB) focus on developing gene therapies for various ailments, the pool of patients whose lives are transformed by this class of treatments is likely to keep growing.
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