From October 6-8, clinicians, therapists and scientists gathered in Warsaw, Poland for the 2016 A-T Clinical Research Conference to discuss ways to improve the care for individuals with A-T.
Highlights from the meeting included:
- An update on the CATNAP Study, a large pediatric neuroimaging study being performed in the UK and co-funded by the A-T Children’s Project. Distinct MRI changes as well as changes in certain brain metabolites were found in children with A-T. Although validation studies are necessary, these changes may be suitable for monitoring disease progression and measuring the effectiveness of therapies in clinical trials.
- A discussion of certain immune parameters that might indicate a worsening disease course, allowing clinicians to identity Individuals with A-T who might need more aggressive supportive care.
- Presentations describing changes that occur in the liver and the types of cancers that occur as people with A-T get older. Clinicians can now begin watching for changes in liver function and recommending surveillance for certain types of malignancies.
- Presentations from clinicians developing new technologies to image the lung that may one day replace chest CT scans for individuals with A-T.
- An update from the Italian company, EryDel, on the planning of their upcoming clinical trial for red blood cell loaded dexamethasone (the EryDex System) in A-T. Final approval from the FDA is needed before this trial can begin.
- An update on cancer in A-T, emphasizing that physicians treating A-T patients with early or limited-stage cancers should take more aggressive approaches intended to cure the disease rather than simply managing it. Additionally, chemotherapy toxicity and supportive care were reviewed; gaps in our understanding and knowledge of cancers in the context of A-T were discussed; and the pros and cons (“who” and “when”) of bone marrow transplantation for individuals with A-T and cancer were debated. Currently, it is felt that bone marrow transplants for people with A-T should be considered on a case-by-case basis and should involve discussions with a team of medical experts.
As one of the conference sponsors, the A-T Children’s Project would like to thank the group of international clinicians and therapists who dedicate their valuable time and work tirelessly to treat and care for people with