OpenZika – PLOS Neglected Tropical Diseases

After several months our article in PLOS Neglected Tropical Diseases is now out from embargo and published. A big thank you to my co-authors Carolina Horta Andrade and Alexander Perryman who have put a lot of their time into the project. I would also like to thank the reviewers for their constructive comments. We have updated this article considerably since we put out the preprint in figshare back in August. As an added bonus we have made some slides which update the OpenZika story. Please tell others how they could contribute their computer time or Android phone (while charging) – every bit helps. We hope to update at some point with molecules we have tested that have been identified by OpenZika.


Zika Homology models paper makes it to PubMed >6 months after “Publishing” in F1000Research

Who would have thought in February that when we submitted our manuscript on Zika homology models to F1000Research it would finally appear in PubMed over 6 months later. This was no where near as fast as our first article on Zika that took a few months to be approved and then appear in PubMed.

What started out as a challenge to model all the proteins in Zika and put it out into the open – turned into a “publishing” learning experience. Thankfully at least the information and the models were in repositories like figshare and F1000Research and not some other journal that would have kept the information closed until acceptance.

F1000Research had a seemingly impossible time finding reviewers – after we suggested what seemed like every possible person in their database and then kept coming back for more. Why was it so difficult to find reviewers for a manuscript on homology models – regardless of if its Zika or not? This is definitely puzzling.

After a month the cryo-EM and crystal structures started to appear in the PDB and in publications. In the six months this paper was not in the PDB we reckon several others have published one or more Zika homology models and had their papers in PubMed before ours – e.g. this and this …one even cited the F1000Research paper before it was “accepted” or in PubMed.

So my take away here is that – yes we got the information out there ASAP on models for Zika and we shared with the community everything, but F1000Research is not very visible on its own. PubMed is important to amplify manuscripts (alongside Google Scholar etc) – we lost >6 months of visibility even though we tried using Kudos to amplify and had 27 share referalls to date (see metrics in figure – as of today it has 2661 views, 707 downloads – so it appears these data are out of date) Kudos on homology paper. Going forward we will be able to track the effect of being listed in PubMed. If only it could have been there when submitted to F1000Research.

Its likely that other scientists will have missed our work and gone off and done the same or similar. I think this could be prevented by making papers visible in PubMed while under review at Open “journals’ etc. This experience suggests that the open publishing, open review model is not there yet – there are still wrinkles to iron out IMHO.





Postdoc Position for Drug Discovery at Collaborations Pharmaceuticals

Been in start up more fore over a year now I need to hire some help and get an office / lab. Ideally I am looking for a PhD or postdoc that wants to learn what I do for a couple of years. The posting below should describe the role. Feel free to connect (no recruiters) – I can only focus on those already in the USA that are legally able to work here. The job is based in the RTP area – office location to be determined.

Job Description

Position title: Postdoc for Drug Discovery

2 year – postdoctoral position is available working directly with CEO to further develop small molecules as treatments for various diseases. We are seeking a highly motivated, independent scientist with either chemistry or pharmacological expertise to join a new drug discovery company. The company is conducting highly interdisciplinary research focused on pre-clinical development of small molecules for neurological diseases, cancer, and infectious diseases. We collaborate widely with academic labs and are funded by multiple NIH grants. The successful candidate will be involved in coordinating external studies and building up our own laboratory capabilities in either pharmacology or chemistry. Must understand the drug discovery and development process and want to help translate treatments to the clinic. Their duties will include direct interaction with collaborators, analysis of data (ADME/PK, Pharmacology) and report/ paper/ grant writing.


Applicant must have PhD in either pharmacology or medicinal chemistry or related topic and be a first author on at least 2-3 publications. This position requires extensive collaboration and problem solving experience. Chemistry and pharmaceutics expertise would be valuable. Excellent oral and written communication skills in English are required. Drug discovery experience in industry would be ideal.
How to apply

Please submit cover letter, CV, and contact information for 2-3 references to Sean Ekins, Ph.D., D.Sc. by e-mail to: collaborationspharma@gmail.com

Start date

Position open immediately



RTP area – currently identifying office and start up lab space.


Employer’s Web Site: http://www.collaborationspharma.com/

Publications: https://www.ncbi.nlm.nih.gov/pubmed/?term=Ekins+s


Progress Through Partnership: The NINDS 2016 Nonprofit Forum

Last tuesday and wednesday (Sept 13-14th)  I was very honored to be an attendee at the “Progress Through Partnership: The NINDS 2016 Nonprofit Forum“. My role was to provide an industry perspective on several panels (alongside Dr. Ronald Marcus, Cerecor). There were a large number of rare disease (patients or parent) advocates, academic scientists and of course a large number of NINDS attendees. Going into this with no previous experience of the meeting I was a little unsure what I could add. But then as I sat there listening I think I had a unique perspective. I sit in an uneasy space as a scientist, a rare disease advocate and also someone that sees how small business can help. I am also impatient, so any hint that the science is not moving or that perhaps something is being missed, then I was on it. Apologies if it comes across as blunt but frankly with many diseases the progress has been too darn slow because of some key opinion leaders basically owning diseases and not having the ability to embrace new technologies or approaches that have been used elsewhere and could make the difference as outcome measures for clinical trials, for example. Below are some simple notes that I made – apologies if I missed anything while also on the panels on day 1, I also happened to live tweet which I have now storified.

Key points from Dr. Walter Koroshetz’s introduction – there were 161 registrants at this 10th non profit forum. He suggested we need to empower patients and power trials. He mentioned a vibrant translational program with Dr. Amir Tamiz. While the average number of grant applications increased by 25%, the average cost of grant is $379k. He mentioned one of the key policy issues being reproducibility. NINDS needs to support basic research (27% of budget). Some of the NINDS programs were mentioned including IGNITE, CREATE , BLUEPRINT as well as specific RFAs such as the Parkinson’s biomarkers program and the accelerated medicines partnership for Parkinson’s Disease.

Panel 1 – Lessons learned and case studies in natural history databases

Steve Kaminsky (International Rett Syndrome Foundation) their foundation saw 4 clinical sites would not be enough for recruiting and therefore built travel clinics (4-11 sites). He described the need for partnerships with NIH programs. Now other rare diseases are in the program, up to 15 sites using the infrastructure. The data created belongs to the nation,  is protected, and the 15 sites have access. Pharma has to go through PI to get access, and the general public can interface with investigators who can study it (what if the patients wanted to study it – he did not seem to allow for this scenario?). The database was part of RDCRN. He mentioned it was too expensive to keep the database going. Over 7000 data fields for 1234 patients has resulted in publications of impact on sleep, effectiveness of drugs, diagnostics, anxiety etc. all from analysis of 12 yrs of data. He suggested the future is through the past and the past is natural history studies.

Michael Shy (University of Iowa) described Charcot-Marie-Tooth and the more than 90 genes identified. The INC RDCRN is in its 8th yr.  They partner with advocacy groups. MDA, CMTA, and groups in the UK and Asia. They have 20 centers involved. He then focused on the CMT neuropathy score, described natural history studies as a science and how with CMT1A, measuring change that’s detectable is important He described frequent calls with researchers (I wondered are the calls with the RDCRN open to any advocacy groups or just close collaborator groups?).

Steve Roberds (Tuberous sclerosis alliance)  owned their database. He discussed the difference between clinic entered vs patient entered data and getting input from scientists for data collection. Getting information from clinical data and electronic medical records was also described. There was some discussion around what happens after the end of a grant and who owns data, the long term sustainability of a database. Such databases present opportunities whether that is partnering with industry, (e.g. he described a collaborations with Novartis), who can fund changes to the database. They can also access without having to build the database. The alternative was Novartis Europe who collected their own data in Europe via a CRO, and learnt what they had so the elements matched with the US database. Steve also mentioned linking to biosamples and how with highly variable diseases e.g. Epilepsy, tumors, etc. this can add value to the database. He also described how custom built databases could be made simpler and facilitate analysis in order to get finding and papers out to in turn encourage utilization. You have to start a NHS sometime and it is a longtime commitment.

Petra Kauffman (NCATS) discussed disease registry vs NHS, and the need for forward looking data management coordination so that surveys can go out fast. She also mentioned that NCATS is launching a Toolkit for rare disease foundations as an Online portal.

Panel 2. Data integration and data management: challenges and opportunities

Paul Gross (Hydrocephalus association) raised questions to be answered and discussed how to engage clinicians and partner with sites that lead in electronic medical/health records. Find clinicians on Epic’s (steering) board. These board clinicians  advocate for inclusion of data sets.

Greg Farber (NIMH) described the national data archive at NIMH which houses data from 130,000 human subjects. Researchers can submit an application and summary data is available to anyone with a browser. Data on  800TB of image data. NINDS has FITBAR database. Holds data funded by other groups and uses common data elements in data dictionary. Every 6 months they request data from NIH grantees and validate data. They use a global unique identifier which is a hash code that can aggregate data on the same subject from multiple labs. Their database makes data potentially discoverable. Greg also mentioned the challenge of professional clinical research subjects and how to identify them and remove from clinical trials. The database is also citable and linked to the literature.

Shawn Murphy (Harvard Medical School) described how to get large sample size for trials by creating a large warehouse in the EHR. 6.7 million patients in such a database relates to 2.5 billion facts. Data in EHR is not very accurate, e.g. you can test for diagnosis using codes and notes in medical records. Finding patients by query construction in the database was also described. He mentioned that there were over 5000 registered users of tool. He had a NIH grant for i2b2 to create a community of developers, the Hive develops new tools. He also described using phenotyping algorithms to define cohorts of treatment resistant responsive. His goal was to create gold standard training sets. Such that the data could be used to build a classification algorithm to predict depression and improve detection. The CTSA at each site forms networks to combine the data and perform big data queries. For example, what is a normal child? Normal MRI provide visual guides and can be used to make new clinical decisions. He mentioned SHRINE and SMART software also.

Tamara Simon (University of Washington) described using databases to look at CSF shunt complications. She used the PHIS database used across 40 children’s hospitals. She mentioned the hydrocephalus core data project which provided a comprehensive, prospective study. This involved detailed data with 15 forms and 413 questions. This also enabled her to address what patient factors were associated with shunt failures, infection etc. PEDSNet database was also mentioned.

Panel 3. Strategies for biomarker identification

Shahshi Amur (FDA) described resources and tools, and different categories for biomarkers , risk, prognostic etc. biomarkers used in drug development and surrogate endpoints. Enablers for endpoints included data quality , assay imaging protocols. She also stated the difference between qualification and validation, To date 13 unique biomarkers have been qualified by the FDA, and 28 submissions. (She did not mention how long to process qualifications – and what it the cost). She did describe natural history data as useful for prognostic biomarkers using strength of evidence.

Other speakers in this session included Dr’s Katrina Gwinn (NINDS), Hao Wang (NINDS) and Petra Kaufmann (NCATS) who discussed agreeing on biomarker terminology and the need to make sure you are going after the right things for biomarkers. The heterogeneity of patients with disease was mentioned. Consortia were described for bringing groups together to look into them,  how to stop reinvention. Standardizing and training staff to decrease noise in order to get the right treatment for the patient was also brought up. Standardization for bio specimens was thought important as well as the need to improve the quality of publications and the data that has to be associated with it. There was some mention of precompetitve efforts for biomarkers and the need overall for teamwork.

NIH101: priority setting, decision-making, NIH basics and discussion

Alan Willard (NINDS) presented on program considerations and the need to fund early stage investigators, concept clearance for proposed solicitations and influencing what they solicit through RFI.  He also alerted us to how people could volunteer for peer review and go to the CSR website to volunteer expertise. There was also some mention of OnPAR as a way to alerting other foundations of applications that were not funded but may be useful for funding by others.

Panel 4. Developing Better Clinical Outcome measures

Ron Bartek (Friedreich’s Ataxia Research Alliance) moderated this session which included Michael Shy (Univ Iowa)  who described outcome measures such as CMTNSv2, CMTPEDs (But did not describe how or if the scores are accepted by FDA). He also described the disability severity index, PCMT-QOL CMT health index and a recent paper that developed an MRI biomarker as published in Lancet neurology in 2016. Carsten Bonnemann (NINDS) again mentioned GAN and the importance of  using outcome measures from other disorders and combining known outcome measures with exploratory measures. Ray Dorsey (Univ Rochester) suggested the need for novel outcome measures.
He illustrated this as we still use a 100 yr old plus method for scoring Parkinson’s disease. He proposed novel sensors, remote monitoring, wearables, implantables etc. as alternatives. Another example was how voice recording can detect Parkinson’s, Parkinsonism and related disorders from 2015. 15,000 people took part in mobile app based study. It is also possible to measure pharmacological improvement in an app. Huntingtons disease and step time was yet another example. He also mentioned the importance of measuring patients function in the home environment. He made an excellent comment at the end of his presentation about some thought leaders embracing technology (and yet there are many others that do not).  Jacob Kean (University of Utah) discussed improving precision in patient reported outcomes, using and adaptive approach for using measures and short form selection. He also described how the NIH invested in PROMIS and the importance of tracking registry participants. Matthew Goodwin (Northeastern Univ) gave a very animated presentation which alerted the audience to the stickiness of technology and how wearables could give very clear kinematic signatures. Examples he used included tracking heart rate in autism, repeated measures which you can see groups clustering in. Also described were differences in hyper vs hypo aroused patients, and in epilepsy you can detect onset with wearable devices that can differentiate tonic and clonic signatures. There was also the potential to get devices to combine information with elements of citizen science for engagement. Some of the drawbacks include the cost of data analysis, in which case sampling might be good to find the signal. There is also the need for researchers that can do the modeling and the health sciences. Increasingly clinical companies are wanting to use mobile technologies.

Other elements are captured in the tweets used in the storify below.


Collaborations Pharma, Inc. And Rutgers Announce NIH Award to Develop Treatments for Tuberculosis

To follow up my last post .. I can now announce some recent funding from NIAID for the TB work with Dr. Joel Freundlich ! Here is the PR –

Fuquay-Varina, North Carolina – The National Institute of Allergy and Infectious Diseases (NIAID) recently awarded $149,388 to Collaborations Pharma, Inc. (CPI) to initiate a partnership with Rutgers aimed at developing a series of compounds for treating tuberculosis (TB), an infectious disease generally affecting the lungs in humans and caused by the bacterium Mycobacterium tuberculosis (Mtb).

One-third of the global population is understood to be infected with TB and the disease continues to kill 1.5 million people every year and to infect approximately 9 million. Despite the availability of effective treatments for the disease, the combined impacts of drug resistance and morbidity of patients co-infected with HIV/AIDS have stimulated research on new quicker acting (less than the current six-month minimum) treatments efficacious against drug-resistant infections that are less toxic when used with anti-retroviral regimens for HIV/AIDS.

“We initially used Bayesian machine learning models to rediscover a class of compounds which seems to have been neglected for over 40 years ago. The compound we found has activity against drug-sensitive TB as well drug-resistant forms” said Sean Ekins, CEO CPI.

“To date my lab has made many analogs of the initial active compound we co-discovered with Dr. Ekins. Our plan is to focus on addressing limitations using computational models developed by CPI to see if we can arrive at a compound with good activity in an acute mouse model of the disease” said Dr. Joel S. Freundlich, Associate Professor of Pharmacology, Physiology & Neuroscience and Medicine at Rutgers University–New Jersey Medical School.

Dr. Freundlich has a chemical biology lab of eleven scientists that utilizes a multi-disciplinary approach to study infectious diseases, with a specific focus on tuberculosis. His lab will aim to identify potential drug candidates as well as the mechanism of action of this antitubercular class. Ultimately their goal is to optimize this compound class to develop a commercially viable new series of antibacterials.

“This work is a wonderful example of our efforts to involve outside companies in our search for novel antibacterials” said Dr. David Perlin, Executive Director, Professor at the Public Health Research Institute Center at Rutgers and Principal Investigator, NIH/NIAID Center of Excellence in Translational Research.

“We are very grateful to NIAID for funding this project as CPI is focused on collaborations with academia so that we can apply our computational approaches to real world applications that can impact research on neglected diseases” said Dr. Ekins.

About Rutgers New Jersey Medical School
Founded in 1954, Rutgers New Jersey Medical School is the oldest school of medicine in the state. Today it is part of Rutgers, The State University of New Jersey, and graduates approximately 170 physicians a year. Dedicated to excellence in education, research, clinical care and community outreach, the medical school comprises 22 academic departments and works with several healthcare partners, including its principal teaching hospital, Newark University Hospital. Its faculty consists of numerous world-renowned scientists and many of the region’s “top doctors.” New Jersey Medical School hosts more than 50 centers and institutes, including the Public Health Research Institute Center, the Global Tuberculosis Institute and the Neurological Institute of New Jersey. For more information please visit: njms.rutgers.edu

About Collaborations Pharmaceuticals, Inc.
Collaborations Pharmaceuticals, Inc. performs research and development on innovative therapeutics for multiple rare and infectious diseases. We partner with academics or companies to identify and translate early preclinical to clinical stage assets. We have considerable experience of preclinical and computational approaches to drug discovery and toxicity prediction. For more information, please visit http://www.collaborationspharma.com/


Gearing up the new company

Well, its been quiet for several weeks as I had a few projects to complete. One was the final report for an SBIR and in addition I have been curating a special edition of Drug Discovery Today focused on Tuberculosis as well as writing grants (I am always writing grants). So lots of writing basically interspersed with some science and collaborations on new projects.

August marks the start of a clean break. I am going to mostly focus on my own company Collaborations Pharmaceuticals, Inc. which started over a year ago but has been in stealth mode. I have been putting together a number of collaborations and writing grants and doing preliminary work. The goal is to use this as an opportunity to address several rare and neglected diseases. It builds on the work Jill Wood, collaborators and I have been doing at Phoenix Nest Inc for Sanfilippo Syndrome. I reasoned what we have done for a single disease could be applied much more broadly. I have been approached by several families with children with rare diseases over the years and I am sure this will continue. What if there was a way to use the Phoenix Nest model to apply to their disease. Namely, provide a means to get STTR grants with collaborator academics working on the disease of interest. So it is pretty simple, as a business model. Identify the diseases, collaborators, write and win the grants and then ultimately license the science. I feel like this is the ultimate experiment. Twenty plus years of collaboration in the industry in various positions have lead to this. Start small, win grants, hire people and build business.

The challenge is continuing to build up Phoenix Nest and also serve as CSO of the Hereditary Neuropathy Foundation which are my major responsibilities as well as my many ongoing collaborations with scientists at many academic groups. This is a good opportunity to evaluate and plan for the future. One of the challenges will be managing a growing number of collaborations and I think this will create new opportunities for software development too. If there is anyone that wants to help me to find excellent academic collaborators then please get in touch. I am always open to new ideas!

I really enjoy the challenge of doing something that is new or a different topic, and in the past decade I have been successful in obtaining small business grants to help build other people’s companies. Now its time to use this ability for something which I started myself. I know it will require dogged persistence and patience (which I don’t have much of). The focus on rare diseases came from a single meeting while that of neglected diseases has built on collaborations initiated over the past 8 years or so and I hope it can build a second sustainable business and create jobs and treatments. As always, I will endeavor to document the process here as I can.



Experience with Reddit Science AMA

Today I took part in a Reddit Science Ask Me Anything (AMA). I had been invited to do this by the nice folks at Springer so I chose rare diseases as my topic and had 100s of questions to answer.

This naturally took far longer than 1hr to answer. Generally a good mix of topics from people introducing their rare disease of interest as well as people asking career advice. I would recommend it as a form of outreach and who knows it will likely lead to new connections. As scientists we should definitely be exploring methods like this to get the word out. Thank you for all the questions!\

(PS – I should add by the end of the day I probably spent 5hrs answering questions..debating how frequently to follow up and check in and respond. The format is not something I have a lot of experience with.)




MM4TB and a trip to a new Miro Exhibit


OIMG_0999 (2)ccasionally I take a bit of a diversion from my usual science focused postings, this one has an artistic flavor.

I am in beautiful Brataslava in the Slovak Republic for a few days for the final More Medicines for Tuberculosis (MM4TB) meeting. Every 6 months for the past 5 years we have had meetings of the members in this EU FP7 funded project working on developing new treatments for TB. To me these meetings have been a wonderful example of global collaboration with over 20 academic or company labs working on various elements from high throughput screening to X-ray crystallography.

This afternoon we had a little break from the science presentations and took a tour of a new exhibit at the Danubiana Meulensteen Art Museum. I was pleasantly surprised by the museum which is very modern and displays an extensive array of modern art from local artists.

The exhibit we came to see is yet to open to the public and it is called “Miro and Cobra” and it runs until the 13th November 2016. We were very honored to have a treat of an exclusive guided tour from the director Vincent Polakovič . I should say the museum itself is a national treasure, but this exhibit is a must see for any Miro fan. I will not spoil it for you but I include only a few photos to whet the appetite!


Having the Miro exhibit is a huge coup for this Museum and the light airy space really does the art a lot of favors, making the colors pop and shining a spotlight on the various sculptures.IMG_0959

My favorite part of the exhibit was the room that replicated Miro’s studio, complete with easels, split cane chairs. It felt like Miro was there and had just walked out for a breath of fresh air in between painting.

So although the MM4TB is coming to a close this trip will be remembered as a highlight. Thank you Katarína Mikušová for organizing the meeting and the magnificent hospitality of your country.

I would recommend a vacation to Bratislava and I will be back in future.

Next we have a little wine tasting before resuming the science tomorrow!   IMG_0962 (2) IMG_0958


BIO2016 and getting Big Pharma to listen to the patient’s voice..

Its hard to believe that a couple of weeks ago BIO2016 was coming to an end. After 5 days in San Francisco my legs were tired, my voice was rough, and I was tired of boxed lunches and eating dinner at crazy irregular times – generally very late. Its a hard life!

I was at the meeting thanks to the nice people at BIO who invited a whole array of patient advocates / disease foundations for the Patient pavilion. Don Gibbons from CIRM did a really nice blog on this and has some photos so you can see Allison Moore from the Hereditary Neuropathy Foundation and I in action. A big thanks to Gautami Inamdar for inviting us.

We used the opportunity to network with companies big and small and find out which ones might have potential therapeutics for Charcot-Marie-Tooth. We also used this as a platform to highlight the upcoming patient centered CMT summit.

Frankly BIO treated all the groups in the patient pavilion like VIPs, we could apply for front row seating to all the key notes, we had our own social event and had a tour of Genentech. This was pretty special, being bused to their site, escorted around a couple of labs, meeting scientists and getting wined and dined. Genentech really rolled out the red carpet. At one point we were in the high throughput screening (HTS) lab and in front of us was an ECHO being used to dispense solutions !  Of course the complete Genentech small molecule collection was also there too, just waiting to be screened in an assay. Many of the patient groups I am sure were thinking, in that 1.5 million or so compounds is a potential treatment for [insert name of their]  disease. Lots of questions were asked here and the scientists involved showed just how passionate they could be about the science. In the social hour afterwards we met several other Genentech employees and heard about what the company was doing. I have to say I know a few friends that work for the company so it was great to learn a bit more about the history behind the company.

After the first day or so we spent time in meetings, attending presentations (mitochondrial diseases stood out here) and walking what seemed long distances around the halls. BIO also gave us the opportunity to give a short presentation on HNF and the research we are involved with and funding.

It was not all rosy though, I happened to meet another rare disease parent at the meeting who has a foundation that has funded some research which had started using an FDA approved drug and shown some promising results in vitro. The parent wanted to meet with someone from the company that marketed the drug and so we went along to their booth. We were met with looks as if we were talking a foreign language. Clearly a huge disconnect with people at this big pharma and rare disease parents/ patient advocates. We went to another company who also has a drug in the same class. Again we were brushed off.. It does not have to be this way. We know from the way Genentech treated us, they listened, they seemed open. It appeared as if some of the companies still have much to learn about the importance of rare diseases.

I also had chance to do a bit more networking with additional rare disease foundations and came away from the meeting thinking what if BIO could organize a much small version of the meeting just focused on Rare diseases and at the same time they brought in some of the big pharma to educate them and build collaborations.. would it work..?








New grants for Phoenix Nest Inc. and LABioMed for Sanfilippo Syndrome treatment development

I can now report some very good news for Phoenix Nest Inc  in our efforts with our wonderful collaborators at LABioMedwe now have a couple of grants to work on treatments for Sanfilippo B and D. 
It would be great if we could find VC or angels that would help us get these treatments to the clinic even faster alongside our work on Sanfilippo C. Please get in touch and help us find more families with this disease
Here is the press release!
Public Release: 

Phoenix Nest and LA BioMed receive over $1.7 million in grant funding

Working together to develop treatments for devastating childhood disease

Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center (LA BioMed)

IMAGE: Patricia Dickson, M.D., LA BioMed lead researcher and director of the institute’s MPS Research Laboratory, will lead the studies seeking new therapies a for devastating childhood disease, MPS III…. view more

Credit: LA BioMed

LOS ANGELES – The National Institute of Neurological Disorders and Stroke recently awarded over $1.7 million in grant funding to Phoenix Nest, Inc. to continue its partnership with the Los Angeles Biomedical Institute (LA BioMed) to research the development of therapies for treating different forms of a devastating inherited genetic disorder, Sanfilippo disease, also known as MPS III.

MPS III disease is a progressive neurological disorder that is generally diagnosed in childhood and can lead to premature death. The new grants are for therapies to combat devastating brain disease due to MPS III types B and D.

Funding for work in MPS IIID is for recombinant enzyme therapy and is a Phase 2 Small Business Technology Transfer (STTR) award, following a successful $223,102 Phase 1 award. Phoenix Nest and LA BioMed were also awarded a Phase 1 STTR grant to develop a novel stem cell therapy for MPS IIIB syndrome. Both treatments have the potential to completely transform the care of children with MPS III disease, as no approved treatments currently exist for any type.

“We are pleased to continue our partnership with Phoenix Nest, Inc. in this important research to help children living with MPS III B and D. After promising results obtained in our first preclinical studies for an enzyme replacement for MPS IIID, we will next look at efficacy in a disease model with a goal of moving towards the clinic as quickly as we can,” said Patricia I. Dickson, MD, LA BioMed lead researcher and director of the institute’s MPS Research Laboratory.

Dr. Dickson has specific expertise in the development of intrathecal enzyme replacement therapies for treating MPS by replacing the missing enzyme through the patients’ spinal fluid.

“We are very grateful to the National Institute of Neurological Disorders and Stroke for continuing to fund this project to find more effective therapies for MPS IIID, which is so debilitating to the patients and causes enormous stress for the families involved,” said Sean Ekins, PhD, chief executive officer of Phoenix Nest, Inc. “The additional funding will be used to manufacture an enzyme for further testing. The research is seeking to develop a therapy that will limit or reverse the neurological damage caused by MPS IIID by delivering an enzyme, recombinant human alpha-N-acetylglucosamine-6-sulfatase (rhGNS), intrathecally to effectively treat the underlying causes of the neurologic symptoms of MPS IIID.”

The National Institute of Neurological Disorders and Stroke also provided funding for development of a treatment for MPS IIIB. “This represents a novel approach to use stem cells to make the enzyme alpha-N-acetylglucosaminidase that is needed to achieve therapeutic benefit,” said Dr. Dickson.

“Phoenix Nest, Inc. is solely focused on Sanfilippo syndrome, and we are honored to continue collaborating with Drs. Dickson, Tsui-Fen Chou, Michelina Iacovino and their teams so that we can develop multiple treatments for MPS IIIB and MPS IIID,” said Dr. Ekins.


About LA BioMed

Founded in 1952, LA BioMed is one of the country’s leading nonprofit independent biomedical research institutes. It has approximately 100 principal researchers conducting studies into improved treatments and therapies for cancer, inherited diseases, infectious diseases, illnesses caused by environmental factors and more. It also educates young scientists and provides community services, including prenatal counseling and childhood nutrition programs. LA BioMed is academically affiliated with the David Geffen School of Medicine at UCLA and located on the campus of Harbor-UCLA Medical Center. For more information, please visit http://www.LABioMed.org

About Phoenix Nest, Inc.

Phoenix Nest, Inc. is a biotechnology company focused on Sanfilippo Syndrome founded by and solely owned by several Sanfilippo Syndrome families. They actively collaborate with academic researchers to fund and commercialize their technologies. Phoenix Nest, Inc. also licenses assets from companies and universities that may have utility for Sanfilippo Syndrome. For more information, please visit http://www.phoenixnestbiotech.com.

For further information, please contact Jill Wood, Co-Founder and Chief Financial Officer, Phoenix Nest, 347-689-2186 jwood@phoenixnestbiotech.com

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