Radicava® approved for treatment of ALS in United States
On May 5, 2017, the Food and Drug Administration (FDA) approved Radicava® to treat patients with amyotrophic lateral sclerosis (ALS) in the United States. The active ingredient of Radicava® is edaravone. Edaravone was originally developed by the Japanese pharmaceutical company Mitsubishi Tanabe for the treatment of acute ischaemic stroke. Subsequently, clinical trials in ALS patients demonstrated that the progression of ALS with edaravone could be delayed. These trials were all carried out in Japan. Since 2015 edaravone is on the market for the treatment of ALS in Japan under the brand name Radicut®. Hereafter, FDA approval was requested for the US market which has now been obtained.
Radicava® is administered directly into a vein by means of a 60-minute infusion. Dosing is done in repeated treatment cycles of 28-days. Infusions are given in 10 of 14 days in the first two weeks, followed by a 14 days drug-free period ‘(drug holiday’). Radicava® may be administered in a hospital or by a health care professional at home.
Treeway is pleased with the approval of Radicava® in the United States. We feel supported by the recognition of the efficacy of edaravone by the FDA. The approval of Radicava® does not interfere with Treeway’s plans for TW001, the treatment we have in development for the oral administration of edaravone.
First ALS Patients Connected Meeting was a success!
In January, the first successful mutual meeting between biotech company Treeway and patient organization ‘ALS Patients Connected’ was organized. Treeway is initiated ‘by patients and for patients’ and this meeting – where we entered into a conversation with one another – is an important aspect. We introduced ourselves and explained our way of working. Although there has not been found a solution to cure ALS yet, we are moving forward in our search for a therapy. One of the things that were highlighted during this meeting is the importance of collecting and sharing ‘real life data’ in order to obtain even better results for the continual development of a therapy. ALS Patients Connected can play an important role here. This meeting is a good example of how we can work closely together and to improve and speed up the research and development of a therapy. For more information about APC:http://www.alspatientsconnected.com
Post-doc position for 3 years
In collaboration with the University Medical Center Utrecht, Treeway is looking for an enthousiastic post-doc who will be supporting extensive bio-informatic analysis of the genetic data obtained within Project MinE. Project MinE (http://www.projectmine.com/) is the world’s largest research initiative devoted to discovering genetic causes, risk factors and disease pathways of ALS by collection of whole genome DNA sequences from 15,000 ALS patients and 7,500 healthy control persons. Integration of these genetic data with publically available biological pathway databases, drug databases and literature results, new biological pathways involved in ALS, new drug targets for ALS and repurposing of existing drugs for ALS might be identified and could as such lead to a new drug therapy for ALS.
Please, click the following link for more information and to apply for this job opportunity: http://www.werkenbijumcutrecht.nl/vacatures/Paginas/postdoc-neurogenetics%20(2017-0081).aspx
Amyotrophic Lateral Sclerosis (ALS) is a devastating progressive, neurodegenerative disease that causes death of motor neuron populations. The loss of motor neurons leads to muscle weakness, difficulty in swallowing, speech and eventual paralysis. Average disease duration is 2-5 years. It is an orphan disease with an incidence of 2 of 100.000, and a life chance of 1:300. Its occurrence is mostly between the age of 40-60 years, although also at other ages ALS is diagnosed. Of all diagnoses, 10% is familial and 90% is ‘sporadic’. For the familial form, different genes have been identified, although the exact mechanism of the cause and the progress of ALS is unknown. Currently, there is only one approved ALS drug therapy, riluzole, but this is prolonging life by only 3 months. Therefore, there is an urgent need for improved drug therapy.
Treeway is a biotech company founded by ALS patients, and our mission is to change the devastating disease Amyotrophic Lateral Sclerosis (ALS) into a chronic disease. Treeway Therapeutics aims to do rational target validation and drug development with a patient-driven fast-track focus and we have established high-potential research and development focus areas up to clinical phase II/III program leading to orphan drug status and early access.
Inez de Greef and Ronald van der Geest were invited to the Drug Discovery and Development Workshop in Florida from 7 to 9 October, organized by the ALS Association. The progress of the second product of Treeway, TW002, will be presented. TW002 is a gene therapy whereby GDNF is delivered with the viral vector of AAV5.
In het artikel ‘Heel de wereld kijkt naar Katwijk’ beschrijft Jan Fens zijn zoektocht naar een medicijn tegen zijn ziekte Hereditary Cerebral Haemorrhages with Amyloidosis – Dutch type (HCHWA-D), beter bekend als de Katwijkse Ziekte.
Uit dit artikel zou ten onrechte kunnen worden afgeleid dat Treeway momenteel edaravone in pilvorm aan patienten geeft. Dat is niet het geval. Treeway verstrekt op dit moment ook geen edaravone in welke vorm danook aan patienten aangezien de werking van edaravone nog onvoldoende is aangetoond.
Het artikel van de heer Fens is op persoonlijke titel en zonder bijdrage van Treeway, samengesteld en gepubliceerd. Treeway heeft vooraf inzage gehad in het artikel, maar het was in dat stadium niet meer mogelijk om nog wijzigingen aan te brengen. Treeway neemt dus ook geen verantwoordelijkheid voor de inhoud en mogelijke onjuistheden die in dit artikel kunnen staan.
Our Founder Bernard Muller and CEO Inez de Greef were interviewed by the FD about Treeway’s mission and ambition. For full article click here.
Bent u ALS-patiënt en wilt u ons helpen onderzoeken hoe we medicatie voor Treeway zo gebruikersvriendelijk mogelijk kunnen ontwikkelen? Vul dan onderstaande patiënten enquete in. Dank u wel voor medewerking!
Great interview with PharmaTelevision and Bernard Muller
Bernard discusses his criteria for entrepreneurship, and how he has turned this into a personal fight against ALS.
Treeway supports Tour du ALS 2016! This year, on June 3rd, our colleagues Ronald and Dirk will climb the famous Mont Ventoux mountain by bike. Will you help our team Treeway Riders to increase awareness and speed up therapy development for ALS? Donate now!
We thank Accenture Digital for organizing the ALS hackathon. It was a very good organized and inspiring day with a lot of enthusiasm!
Clusters of patients with similar disease progression have been identified in advance. While during the event, the participating teams were asked to predict these clusters. Predicting these groups can benefit the field with: patience comfort, better understanding of drug effects and better drug trial management. Out of the 40 participants, 7 teams were formed who took on the challenge to analyze the roughly 38 million data points. After the 8 hours of hard work, the teams with the best algorithm and the most creative insights were both rewarded with prizes. This eventually led to many creative and insightful ideas and results on which ALS research can build further.