Graves’ disease (GD) is the most frequently occurring form of hyperthyroidism. It affects around 3% of women and 0.5% of men over a lifetime. It occurs most commonly in patients in their forties and fifties. Working-age women are the largest group affected. GD is caused by the production of autoantibodies that stimulate cell-surface thyrotropin, or thyroid stimulating hormone receptor (TSHR), to release more thyroid hormones.

There have been no new treatment options for hyperthyroid patients with GD for more than 60 years. At present, antithyroid drugs (ATD), mostly carbimazole, are the usual first-line treatment. Radioactive iodine and total thyroidectomy (thyroid removal) are reserved as second-line treatment options in most UK health settings. Given ATDs induce drug-free remission in only 50% of patients, and both radioiodine and thyroidectomy result in lifelong hypothyroidism, there is no ideal treatment for GD and a new approach is urgently needed.

New potential drug to treat Graves' disease (K1-70)

This study has now stopped recruiting for patients. Thank you to everyone who took part. The study has enabled the researchers to reach more patients. We will report on the results of the trial when we receive them. 

This phase I study is investigating a potential new drug, K1-70, to treat patients who have Graves’ disease, and patients who would benefit from controlling Thyroid Stimulating Hormone Receptor (TSHR) activity. Patients who were diagnosed and treated for thyroid cancer more than five years ago may also be eligible to take part in the trial.

A Safety Review Committee found the drug to have been safe and well-tolerated in all the patients treated so far. The company is now actively recruiting for their final cohort so please contact the site urgently, before the end of September 2020.

Travel to and from the study site is provided from a patient’s chosen location, as well as accommodation for patients traveling from far, with COVID-19 safety considerations in place.

If you would like some more information or are interested in taking part, please contact the Volunteer Services team at the Medicines Evaluation Unit on 0800 655 6553 and quote study MEU 15/304.

Project DAVIAD

This project is not currently active

A company called Apitope has been working on a new treatment for GD that involves antigen-specific immunotherapy. This is a treatment which seeks to reinstate immune tolerance to the target (in this case TSHR). This new treatment called ATX-GD-59 is a combination of two peptides and is based upon synthetic copies of the parts of the TSHR that the immune system recognises as a target (epitopes). 

In 2018, Apitope completed its first clinical study, partially funded by the European Commission, using ATX-GD-59 in patients with previously untreated mild to moderate Graves’ disease.  Twelve participants (11 female) were given 10 doses of ATX-GD-59 intradermally (injections into skin) over an 18 week period. Patients were monitored to ensure that the drug was safe and to observe if there were any changes in the serum-free thyroid hormones and TSHR antibodies.

From the ten participants who finished the course of treatment, seven (70%) showed an improvement in their thyroid function (levels of free thyroid hormones decreased) and the drug was safe and well-tolerated. The 70% improvement in thyroid function was impressive and Apitope is currently seeking further investment to conduct additional clinical studies.

Future studies may also include other groups of patients with GD particularly young patients; patients with severe GD and those with thyroid eye disease (Graves’ orbitopathy); to realise the full therapeutic potential of this new treatment.

Importantly, if immune tolerance can be restored, the underlying thyroid endocrine function is likely to remain intact for many years offering the potential for a more effective treatment of Graves’ disease.

For further details about this study follow this link. 

A short video about GD and this study is also available on our YouTube channel.  

This project was funded from the European Union's 7th Framework Programme for research and innovation under Grant Agreement Number: 602779