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Scientific News

FARA funds research progress

In this section, you will find the most recent FA research publications, many of which are funded by FARA, as well as information on upcoming conferences and symposiums. You can search for articles by date using the archive box in the right hand column. To locate FARA Funded or Supported Research, click the hyperlink in the right hand column. You may also search for specific content using key words or phrases in the search button at the top right of your screen. Please be sure to visit other key research sections of our website for information on FARA’s Grant Program and the Treatment Pipeline.



Randomized, double-blind, placebo-controlled study of interferon-γ 1b in Friedreich Ataxia

In vitro, in vivo, and open-label studies suggest that interferon gamma (IFN-γ 1b) may improve clinical features in Friedreich Ataxia through an increase in frataxin levels. The present study evaluates the efficacy and safety of IFN-γ 1b in the treatment of Friedreich Ataxia through a double-blind, multicenter, placebo-controlled trial. Ninety-two subjects with FRDA between 10 and 25 years of age were enrolled. Subjects received either IFN-γ 1b or placebo for 6 months. The primary outcome measure was the modified Friedreich Ataxia Rating Scale (mFARS). No difference was noted between the groups after 6 months of treatment in the mFARS or secondary outcome measures. No change was noted in buccal cell or whole blood frataxin levels. However, during an open-label extension period, subjects had a more stable course than expected based on natural history data. This study provides no direct evidence for a beneficial effect of IFN-γ1b in FRDA. The modest stabilization compared to natural history data leaves open the possibility that longer studies may demonstrate benefit.

Read the entire article HERE

Therapeutic Prospects for Friedreich's Ataxia

Friedreich's ataxia (FRDA) is a progressive disease affecting multiple organs that is caused by systemic insufficiency of the mitochondrial protein frataxin. Current therapeutic strategies aim to elevate frataxin levels and/or alleviate the consequences of frataxin deficiency. Recent significant advances in the FRDA therapeutic pipeline are bringing patients closer to a cure.

Read the entire article HERE

StrideBio and Takeda Sign Collaboration and License Agreement to Advance Novel Gene Therapies for Neurological Diseases

DURHAM, N.C., March 28, 2019 /PRNewswire/ -- StrideBio, Inc, a leading developer of novel adeno-associated viral (AAV) based gene therapies, today announced the signing of a collaboration and license agreement with Takeda Pharmaceutical Company Limited (Takeda) to develop in vivo AAV based therapies for Friedreich's Ataxia (FA) and two additional undisclosed targets. These programs aim to utilize novel AAV capsids developed by StrideBio to improve potency, evade neutralizing antibodies and enhance specific tropism to tissues including the central nervous system.

"We are very excited to partner with Takeda given their expertise and commitment to developing treatments for patients with neurological diseases," stated Sapan Shah, Ph.D. Chief Executive Officer, StrideBio, Inc. "We look forward to working together to bring transformative and novel AAV-based gene therapies to patients, while continuing to validate and expand StrideBio's platform, manufacturing capabilities and pipeline."

Read the entire article HERE

Patient reported outcomes in Friedreich's Ataxia after withdrawal from Idebenone

Idebenone is a potent antioxidant which has already been evaluated in several clinical trials in FRDA, with reports of symptomatic benefit but inconclusive objective results. Following patient consultation on design, this group completed a treatment withdrawal study to establish whether patients could correctly determine their treatment allocation to placebo or idebenone. Their aim was to capture subjective experiences of symptoms such as, for example, fatigue, which can be difficult to measure with questionnaires or semi-quantitative scales, particularly in chronic, slowly progressive conditions. Patients taking idebenone for at least 12 months as part of the open-label MICONOS Extension Study were randomized to receive either placebo or idebenone continuation for two-month treatment cycles. The primary endpoint was patient assessment of treatment assignment. A total of 29 patients were randomized, forming the idebenone group (n=16) and the placebo group (n=13). No significant differences were detected between the idebenone and placebo groups on assessment of treatment assignment or early study withdrawal. A small but significant difference in ataxia rating scale scores was detected between treatment groups when considering ambulatory patients only. This study provides no data to suggest that FRDA patients could correctly determine their treatment assignment over a 2-month period. We hope that this study design will help inform future trials so that patients' experiences of symptoms are more reliably measured.

Read the entire article HERE

Assessment of cell-free levels of iron and copper in patients with Friedreich's ataxia

Insufficient frataxin levels in FA patients lead to iron and copper deposits in the brain and cardiac cells. 25 confirmed FA patients were included in this study. The total iron and total copper concentrations were measured in blood plasma both in patients and age, sex matched healthy controls. The iron levels in plasma of FRDA patients were found to be significantly decreased as compared to healthy controls. A similar trend was observed in case of plasma copper levels in FRDA patient as compared to controls. Present results clearly show abnormal distribution of extra-cellular iron in FRDA patients, which is in accordance with the well established fact of intracellular iron overload, which is a key feature of the pathogenesis of this disease. This can be of importance in understanding the pathophysiology of the disease in association with frataxin/iron. It appears that intracellular sequestration of trace metals in FRDA patients (due to low frataxin) results in their sub-optimal levels in blood plasma (extra-cellular) an observation that can find prognostic application in clinical trials.

Read the entire article HERE

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