Categories: Featured , In The Pipeline

120 PALS to Enroll in Retigabine Trial

clock April 21, 2015

A group of collaborators announced their intent to launch a clinical trial in 12 academic sites on the anti-epileptic drug, Retigabine (aka Ezogabine), in people diagnosed with ALS. The sites will be chosen by the Northeast ALS Consortium, and include sites in California (3), Massachusetts (2), Michigan, North Carolina, Maryland, Georgia, New York, Florida and Arizona. Screening for enrollment should be expected to begin sometime before the end of 2015. The primary goal of this research project is to study the effect of retigabine on upper and lower motor neuron physiology in ALS patients, however researchers have outlined a number of secondary outcome measures as well such as safety etc.

At the end of 2014, during a scientific meeting in Brussels, Kevin Eggan, Ph.D., who led the team doing the preclinical research behind the selection of this drug, reported that the trial will enroll up to 120 people between the 12 sites and that approximately 1/3 of PALS will be given placebo in the trial. According to Brian Wainger, M.D., PALS enrolled and selected for the active compound arm will be split into two cohorts, one receiving 600 mg and the other 900 mg of Retigabine. 

In a webinar held shortly after the announcement, Wainger told PALS to expect to be in the study for at least 10 weeks. To be considered for enrollment, PALS must meet certain enrollment criteria such as having a possible, probable or definite ALS diagnosis and symptom onset occurring no more than then 36 months ago.  There are several specific exclusion items, most important perhaps to PALS with bulbar onset is that to enroll in the study a PALS must be able to swallow the retigabine pills throughout the study. Other examples of exclusion include the presence of a feeding tube or treatment for a serious cardiac issue at time of screening. Full study information is available in the global ALS clinical trial database.

As mentioned in that report from December, researchers at Eggan’s lab intend to collect skin samples from enrolled PALS from which they can create patient derived stem cells, sometimes referred to as induced pluripotent stem cells or more simply, iPSc.  Researchers plan to then test those iPS lines to determine if they could be used in future trials to determine whether or not a specific PALS would potentially benefit from this drug. The group working on this clinical trial include Harvard Stem Cell Institute, where Dr. Eggan is based, Massachusetts General Hospital Neurological Clinical Research Institute, GlaxoSmithKline, and the ALS Association. 

This trial, along with all others, regardless of who funds them or where they are located, are listed and tracked by staff at the ALS Therapy Development Institute. You can view that global database and subscribe to receive email updates on trial launches, etc, by clicking here.

Bottom Line:

The Retigabine trial is NOT currently enrolling PALS. The ALS Therapy Development Institute has spoken about this drug and the trial multiple times, and we encourage people to review comments from our science team members, Drs. Lincecum and Perrin and others in recent webianrs on the Brussels meeting and Clinical Trials in general. In addition, there is an active discussion thread on this proposed clinical trial on the ALS Forum, in which our CEO, Dr. Steve Perrin provides his individual comments on the drug and trial.

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Categories: Featured , Postcards

Neuralstem Phase 2 Clinical Trial Results Annouced

clock March 13, 2015

Neuralstem recently announced via press release results from a Phase 2 clinical trial of their fetal-derived stem cell treatment, NSI-566. According to their statement, only one person in the 15-person study failed to tolerate the surgical procedure involving the injection of up to 16 million stem cells directly into the spinal cord. The primary endpoint defined for the study was safety. From early analysis of the data, the company reports that endpoint was met. An earlier clinical trial conducted in ALS patients also reported similar results regarding safety.

Nerualstem also included early analysis on the effects of their stem cell treatment on the progression of ALS.  According to their press release, nearly half of the people in the trial responded to the treatment in a positive way. A positive response is defined by Neuralstem as a positive change in the rate of progression as measured by ALSFRS-R or a positive change in grip strength tests. No further details were provided about this cohort, which they term the responder group. The other half of the patients in the trial, termed the nonresponder group, saw a negative change in rate of progression as measured by ALSFRS-R or a negative change in grip strength tests.  Nowhere in the release did they describe an objective measure that could differentiate a responder from a non-responder before enrolling a patient in the trial. That work is ongoing according to several of the investigators quoted in the company's press release.

While the trial was not intended to measure efficacy, the data in the press release seemed to suggest that Neuralstem wanted to communicate on that topic. There was no placebo arm in this study and no attempt to compare the data to a standard of care or placebo arm was made in the press annoucements. All patients were given some amount of modified stem cells which the company hoped would engraft into the spine, replacing cells lost to the disease and providing support to remaining motor neurons, however specific dosing for each patient or across the proposed reponder and nonresponder groups were not provided in the press statement. That is not a surprise and Neuralstem stated that it planned to produce more data later in the year at scientific conferences for example.

One analyst who wrote on the Nerualstem press release, compared the reported ALSFRS-R changes in the Phase 2 clinical trial to a historical dataset, PRO-ACT. According to that person's analysis, when the data from all participants are analyzed together against the historical control group, it indicates the treatment may have had an overall adverse effect on disease progression. Such use of historical controls in lieu of placebo control arms in drug trials remains a controversial topic. Principal investigator, Eva Feldman, MD, PhD, states clearly in the company’s release that she intends to move the proposed treatment into a larger controlled trial, which could be interpreted as one including a placebo or standard of care arm. She suggested that the trial may open for enrollment as early as this summer.

It is the opinion of the ALS Therapy Development Institute that the Phase 2 clinical trial was not designed to measure the efficacy of NSI-566. However, we are encouraged by this additional trial finding the treatment and treatment procedure to be safe and tolerable, in general, in people diagnosed with ALS. We believe that Neuralstem should continue its efforts with this treatment, including the organization and execution of additional clinical trials.

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Categories: Featured , In The Pipeline

Ibudilast Clinical Trial Halfway Enrolled

clock February 27, 2015


Last week, MediciNova announced that they have enrolled 30 PALS in their Phase 2 clinical trial of MN-166, also known as Ibudilast. The trial is enrolling people diagnosed with ALS at a single site in Charlotte, North Carolina, USA.  These types of announcements are common in clinical research programs.  MediciNova and the study’s principal investigator, Benjamin Brooks, M.D., are seeking a total of 60 PALS for participation. At the pace currently, it is estimated the trial may become fully enrolled by the end of 2015 and results from the study should be available by the middle of 2016.

Ibudilast (MN-166) is thought to help motor neurons stay alive by modifying the presence of amino acids, called cytokines, which cause motor neurons and supporting cells to become puffy or inflamed. Tamping down the expression of certain pro-inflammatory cytokines is an approach of great interest in the ALS research field. For more information on this clinical trial, including links to relevant research papers and comments from people in the clinical trial itself, click here. 

The ALS Therapy Development Institute encourages PALS and their families to become informed about the current ALS clinical trials and research. We provided an overview of 14 different clinical trials during a recent webinar with our CEO, Dr. Steve Perrin earlier this month. You can access that webinar by clicking here.

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Categories: In The Pipeline

NurOwn Phase 2 Clinical Trial Update

clock February 3, 2015

It was recently announced via press release by BrainStorm Cellular Therapeutics that their proposed treatment for ALS, NurOwn, will continue to be investigated in ALS in the United States through a Phase 2 clinical trial. According to the latest update provided by BrainStorm on the status of the clinical trial, the ALS Therapy Development Institute lists this trial as “currently recruiting” in its global ALS clinical trial database.

The announcement comes as a result of the trial’s Data and Safety Monitoring Board (DSMB) recommendation for the continuation of the study.  This is not uncommon, highlighted by a similar announcement from AB Science regarding their compound masitinib.  It is one of the jobs of a DSMB to review data continuously throughout the trial they are charged with monitoring, and to make a recommendation for its continuation or cancellation based on that data. This announcement should not be interpreted as any indication this treatment is effecting ALS disease progression in either a positive or negative way. It should only be considered a sign that the DSMB believes that the trial should continue forward.  It should be expected that a final report of the trial’s results will be known only after the trial is completed, which shouldn’t be expected for another year based on the timelines outlined by BrainStorm previously.

What is NurOwn?

NurOwn may help keep motor neurons healthy in people with ALS by boosting production of GDNF, a neuroprotective substance which promotes their survival and growth. In this potential treatment strategy, adult stem cells from the patient's own bone marrow are reprogrammed into GDNF-secreting astrocyte-like cells and reintroduced. NurOwn can likely be administered without anti-rejection medicines. Earlier clinical trials have taken place on this approach in Israel.  Investigators are exploring both intra-muscular and intra-spinal administration of their stem cells in people diagnosed with ALS in a Phase 2 clinical trial currently enrolling in the United States. 

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Categories: From The Bench

Newron Announces Phase 2 Clinical Trial of sNN0029 (VEGF)

clock January 27, 2015

Earlier this month, Newron Pharmaceuticals announced that it would soon execute a Phase 2 clinical trial of its VEGF treatment known as sNN0029.  The announcement comes shortly after one of the lead investigators, Philip Van Damme, PhD, reported the results of the Phase 1 clinical trial during the International ALS/MND Research Symposium in Brussels held in December 2014. The Phase 2 clinical trial announced by Newron is not open for enrollment at the time of publication of this statement.  As soon as it is, we will update our clinical trial database which provides email updates upon subscription.

VEGF is thought to play a role in the health of motor neurons, and the company NeuroNova began a clinical trial of their VEGF compound sNN0029 to in 2008. Newron Pharmaceuticals acquired NeuroNova in December 2012. 

Earlier preclinical studies conducted in SOD1 rats with the compound, and others in SOD1 mice, where both quite small but produced modest improvements in survival overall. These results provided the company enoughinformation to launch their clinical research efforts. 

The Phase 1 clinical trial of sNN0029 included three different doses of the compound, as well a placebo arm.  This multi-arm clinical trial is now completed.  In total there were 19 PALS enrolled in the study, which included two different cohorts, one placebo control (N=10) and the other not (N=8).  In the placebo controlled cohort, four PALS received the high dose (2ug/day) and three received a lower dose (0.8 ug/day) and were compared to three additional PALS given a placebo.  The other cohort looked at a lower dose as well (0.2 ug/day) in 2 PALS, 2 at the middle dose and 4 at the high dose.  During his presentation in December, Van Damme was clear to the audience that the study was in no way powered to measure efficacy, but that the data suggested clearly that sNN0029 was safe and tolerated in all PALS in the trial.

It is interesting to note that 66% of the trial’s participants were male, and on average the onset age of enrolled PALS was 48 years old.  During the post-presentation discussion, audience members asked Van Damme if he thought that a higher dose of VEGF would be tolerable, which he responded he thought perhaps but that any follow up trial would likely use the doses informed on in the early trial.  The announcement from Newron recently reports that PALS enrolled in the Phase 2 clinical trial may receive does up to two times greater than those given in the earlier trial.

For More Information:

·         About sNN0029

·         Newron Press Release

       ·         Recap of Van Damme’s Presentation on sNN0029 during 2014 International ALS/MND Research Symposium

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Categories: Spotlight

Genervon Clinical Trial Update

clock January 15, 2015

It was announced, via press release, that a compound being pursued by Genervon Biopharmaceuticals LLC may have been effective in improving the ability of one person with ALS to speak and swallow.  It is paramount to note that this report is from a single patient, who was exposed to the drug GM6 though a compassionate use patient trial.   Further, this data alone is unlikely to be sufficient evidence for FDA approval of GM604 (aka GM6) as an effective therapy for ALS. Further analysis of GM6 is highly likely to occur both in and out of clinical trial settings.

The PALS participating in the compassionate use trial is male and was diagnosed with ALS nearly 10 years ago. According to Genervon, he has been a quadriplegic and on a ventilator since 2008. The purpose of this study was to gather data from later stage ALS patients and compare it to data from a phase 2 trial done in early stage ALS patients. The company conducted a number of tests on patients’ cerebrospinal fluid (CSF) to attempt to determine if their compound had any measurable effect on proposed biomarkers of ALS thought to be found in CSF, including SOD1, Cystatin C, and tau.

Earlier clinical trials of Genervon include a Phase 1 in healthy volunteers and a Phase 2 trial on 12 people with ALS.  The double blind placebo controlled trial was conducted over a period of 12 weeks with PALS receiving the drug (or placebo) intravenously during the first two weeks only, with follow up visits after that. CSF samples were collected before being given drug (baseline), at completion of treatment program (week 2), and at the end of the observational period (week 12). This trial’s main objective was to measure changes in biomarkers found within CSF overtime and to determine if GM6 was safe and tolerated well by PALS.  Results from this study have not yet been published by Genervon, however it put out a press release in October 2014 claiming that they saw a “statistically significant” effect on both clinical and biomarker data in both ALS and PD clinical trials. 

It is the opinion of ALSTDI that this trial was too small and too short to statistically measure an effect on disease progression.  However, we believe that Genervon should continue its effort with their compound, including the organization and execution of additional clinical trials.

What is GM6?

Described as an “endogenous signaling master regulator,” GM6 seems to be a unique compound created to regulate a specific aspect of cell health, specifically motor neurons. There are many things which motor neurons require to be healthy and part of a vibrant system, including a well-regulated abundance of different proteins called neurotrophins or neurotrophin factors. These include brain-derived neurotrophic factor (BDNF), glial derived neurotrophic factor (GDNF), and mononeurotrophic factor (MNTF) among others.  GM6 is proposed as a regulator of MNTF.  MNTF is believed to play a role in nearly every aspect of motor neuron development; including initial differentiation, maintenance, survival, axonal growth, and inervation of axons into muscles. Genervon believes that GM6 regulates, directly or in part, the expression of more than 4000 genes in the body, including some which are involved in ALS biology.

Helpful Links:

About GM6: Statement on Phase 2a Clinical Trial:

Genervon Press Releases: 

Washington Post "ALS patients press FDA for quick access to controversial biotech drug"

ALS Forum Thread:

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Categories: In Translation

Masitinib Phase 3 Clinical Trial Update

clock January 15, 2015

It was recently announced via press release from AB Science that their drug masitinib will continue to be trialed in ALS.  Currently, more than 200 people are enrolled in the trial, which is taking place in a single center in Spain. The announcement comes as a result of the trial’s Data and Safety Monitoring Board recommendation for the continuation of the study.  This is not uncommon.  It is one of the jobs of this board to review data continuously throughout the trial and to make a recommendation for its continuation or cancellation based on that data. This announcement should not be interpreted as any indication that masitinib is effecting ALS disease progression in either a positive or negative way. It should only be considered a sign that the Board believes that the trial should continue.

What is Masitinib?

Masitinib aims to slow progression of ALS by reducing inflammation. A kinase inhibitor, masitinib blocks mast cell-mediated degranulation, the release of cytotoxic substances that might further damage the motor nerves. Marketed by AB Science under the name Kinavet, masitinib is currently being used to treat certain cancers in dogs.  It is important to note that masitinib is also in a large Phase 2b/3 clinical trial for Multiple Sclerosis.

What preclinical evidence is there for Masitinib?

There have been preclinical experiments on masitinib reported, however ALSTDI has not sought to independently reproduce those findings. There were some challenges with the preclinical reports, including that at least one of the studies was done in female mice alone.  The full data sets on these preclinical experiments haven’t been published, so it is challenging to comment fully on them.

Helpful Links 

FMI about Masitinib:

FMI about the Clinical Trial:

Click Here to Read the Press Release from AB Science

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Categories: Meeting Report

20th Alliance meeting focuses on promoting research and membership

clock December 10, 2012

Gudjon Siggurdson at ALS Therapy Development Institute November 2012

Leadership. Sigurdsson, seated, led a group from the Reykjavik-based International Center for Research on Motor Neuron Diseases on a tour of ALS TDI in Cambridge prior to flying to Chicago

“Stay in touch with patients, we are the specialists in this condition,” said Gudjon Sigurdssonchairman and PLS patient from Iceland, during the 20th annual meeting of the International Alliance of ALS/MND Organizations held in Chicago on December 2 & 3. 

Completing a four-year term at the helm of the nearly 70-member alliance, Sigurdsson gave these and other parting words of advice to advocacy groups working to provide better care and treatments for people with ALS. Jeffrey Dietch, PhD, director of the ALS Hope Foundation was elected as the next chairman of the International Alliance.

The organization has grown over the last number of years, and welcomed new members this year from Peru, Russia and Latvia.  Independent member and nurse Kathy Mitchell, and others, have been travelling around the globe to identify potential new members and determine their nation’s pALS’ needs.

Don’t wait for the funding, do what you love,” said Mitchell, “the money will come.” She cautioned that those like her who seek out advocacy groups need to have flexible goals, measure outcomes and continuously be responsive to the unique political and cultural climates of each nation.

Brainstorming communications

“Israel may be the only country in the world where once a person goes on a ventilator it is illegal for them to be removed from it,” said Efrat Carmi, CEO of IsrALS, the only ALS-focused organization in Israel.  Earlier in the two-day meeting, Carmi had a warning for the US-based organizations to get ready. 

Several years ago, IsrALS issued a small grant to researchers to investigate the potential for stem cells as a therapy for ALS.  The grant, in part, led to the launch of clinical trials of BrainStorm Cellular Therapeutics’ NurOwn.  While excited about the promise of stem cells as a treatment for ALS, Carmi reported key challenges.

“Patients and families are very aggressive.  They want it.  They want to know who they can bribe to get it,” said Carmi.

With a potential Phase II trial of BrainStorm coming to the greater-Boston area in the United States early next year, Carmi advises her American colleagues to develop an open line of communication with BrainStorm and ALS patients well in advance.  

“There are 12 patients in the trial, and the company reports efficacy.  But I know these families and they don’t report efficacy to us.”

From Russia with love

Gleb Levitsky, MD

Reemergence. Gleb Levistky, MD, and colleagues at the Russia ALS Charity Foundation aim to create the first sustained research and care system for patients in Russia since ALS was first documented there in 1889.

The first case of ALS/MND is Russia was reported in 1889.  However it wasn’t until 2006 that national guidelines for the diagnosis and care of ALS patients were established according to the Russian Charity ALS Foundation Gleb Levistky, MD PhD. 

As many as 7,000 people are living with ALS in Russia.  But there are only 3 NIPPV devices available for ALS patients in Russia today according to Levistky.  His organization has helped provide them to 14 people with ALS over the last six years. 

Levistky hopes to create awareness and encourage greater support for people with ALS in Russia.  In 2011, he helped to organize the first ALS certification course for neurologists and worked with Mitchell and others to advocate for assistance from the Russian Ministry of Health.

Raising Peru

The incidence rate may be higher for those living in Peru than in other places in the world according to ELA Peru’s Gabriela Zarate.  Nearly 5 out of every 100,000 citizens have the disease. ELA Peru, launched in 2009, aims to improve the quality of life for the approximately 1,500 PALS in the small Latin American nation, no matter where they live. 

This includes Jamie Ramirez, who in the early 1990s, a convicted terrorist sentenced to prison for at least 25 years.  Almost 17 years in prison, he was diagnosed with ALS.  He requested a shortened sentence to receive treatment, which he did twice, both times denied.  A third attempt was pending when Rameriz passed in October 2012. 

Ramirez’s struggle helped ELA Peru raise awareness of ALS in Peru according to Zarate.

Other news

Rod Harris, Executive Director of MND Victoria, a member of MND Australia, reported that the country is soon to develop a new entitlement system for the more than 400,000 disabled including nearly 1,500 PALS.  The proposed program however would only provide coverage for people under 65 years old. 

Next year, the International Alliance will meet in Milan, where it met 10 years ago.  In 2014, the Alliance will meet for the first time in Brussels, Belgium, hosted by ALS Liga Belgium.  Many in the Alliance expressed excitement for this meeting and the opportunity to see the soon to be constructed “Care Center Middelpunt” on the North Sea.  This facility will house 20 PALS, providing them with care and access to state-of-the-art technology at no cost.  The center is scheduled to open in June 2013.


About 100 delegates participated in the 20th meeting, representing several dozen different nations.  More than 20 patient health professionals and patient advocates presented their experiences.  Incoming chair Jeffrey Deitch of the ALS Hope Foundation presided over the meeting, and incoming Alliance coordinator Rachel Patterson took the official minutes.  An audio recording of the two-day meeting will be available on the International Alliance’s website,  


NOTE: The ALS Therapy Development Institute is an Associate Member of the International Alliance of ALS/MND Organizations.

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Categories: Meeting Report

Momentum there for ALS Treatments According to Panel at TDI Summit

clock November 13, 2011

With momentum today in the lab and in the clinic, now may be the time for real outcomes in the effort to alter the progression of ALS. That was the message from the panel of experts during the afternoon Leadership Panel at the 7th annual Leadership Summit organized by the ALS Therapy Development Institute. The first year of such a panel included sparkling debate, quality analogies and clear advise for the nonprofit organization focused on developing effective treatments for patients today. The entire two-hour "Leadership Panel" discussion was recorded and is available to view online at

Henri TermeerHenri Termeer

George ScangosGeorge Scangos

Douglass OnsiDouglass Onsi

“Do what you are doing, enhance it and make it sustainable from a funding point of view. I think you will get a true effect on this space at this point because I think that at this point this space is particularly conducive for real progress,” said Henri Termeer, former chairman, president and chief executive of Genzyme Corporation. Genzyme has developed several therapeutics for rare and orphan diseases. Termeer, pictured to right at top, stepped down recently after Sanofi purchased the 30 year old biotechnology company.

During a period of questions from the audience, a caregiver for a PALS named Bob who can no longer speak due to the progression of the disease, asked CEO of Biogen Idec, Dr. George Scangos, why his company limited enrollment into their Phase III Clinical Trial "Empower" to people diagnosed with ALS relatively recently. The caregiver relayed that Bob felt that he was being "punished" for surviving longer than others diagnosed with the disease. Scangos, pictured to the right in the middle, empathized, saying that it was a very difficult decision for Biogen when it came to determining the trial design. He reported that after a review of the process and timeline, that it would be faster to determine efficacy if they continued with the design that had been used by Knopp Neurosciences and the NEALS Consortium during the Phase II trial of the experimental treatment. Biogen licensed the molecule from Knopp earlier this year and enrolled more than 800 PALS in less than 4 months into the trial. PALS within 24 months of overt symptom onset were targeted for enrollment, which is the same criteria used in the Phase I and Phase II clinical trials by the University of Virginia and Knopp prior to Biogen licensing the compound from that company. He commented that Biogen is committed to the ALS community and walked through their disease focused pipeline, which includes a soon to begin enrolling FDA-required second Phase III trial of dexpramipexole (Endeavor) as well as several other promising leads in the biotech-leader's preclinical pipeline.

Representing the venture capital and venture philanthropy worlds, Douglas Onsi, Managing Director of Healthcare Ventures provided clear advice to the more than 200 advocates, patients and caregivers in the room at the Hotel Marlowe: you have the power to influence investment. He encouraged audience members to advocate to groups and projects they supported and to never back down. Onsi, picture to the right at the bottom, commented that venture firms often rely on experts at TDI and similar organizations for advice and information about fields they are interested in investing it. In addition to helping to create new companies through his venture work, Onsi is a member of the MS Society's Fast Forward board of directors, where he helps to raise capital and make strategy investments in promising research for new treatments for multiple sclerosis. "You all are the real venture capitals," said Onsi to the audience of mostly PALS and CALS.

During the closing remarks, Dr. Christopher Austin, Science Director of the NIH National Center for Translational Therapeutics, suggested that in these financially challenging times, where there is momentum everywhere but little cash, all the players need to come together. "It's like this, when a slime mold runs out of food it groups together with other slime molds around it to create a totally new entity in order to survive and grow. We need to all start doing the slime mold."

Other participants included Dr. Steve Perrin, CEO/CSO of ALS TDI, as well as Robert Blum, President & CEO of Cytokinetics, which recently announced the start of Phase IIb enrollment for its own clinical trial in ALS with a drug, CK-506 (link to clinical trial page), targeting skeletal muscle.

"ALS TDI is a highly efficacious effort. Why tweak this? I am extraordinary impressed," said Termeer reflecting on what advice to give ALS TDI. He closed in reiterating that he believes that the time may be right and momentum is there. "I have learned so much today that I did not know before."

To facilitate the continuation of that momentum, the panel's moderator, Dr. Myles Axton, is planning on publishing an editorial review of the advice received and outcomes from the panel in Nature Genetics later this month. To view a recording of the panel discussion, click here

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Categories: Meeting Report

Highlights from the 3rd Annual Partnering for Cures, a FasterCures Meeting

clock November 13, 2011

Priming the Pump: Filling the Therapeutic Pipeline

Earlier this month, the 3rd annual Partnering for Cures conference was held in New York City. This event, which began in 2009 is organized by FasterCures, a “action tank” that works to improve the medical research system in order to speed up the time it takes to get important new medicines from discovery to patients. ALS TDI is a member of the TRAIN program at FasterCures, which is a subsidiary of the Milken Institute.

To being the conversation, an opening plenary focusing on the convergence of stakeholders in the funding of research. According to conference organizer and FasterCures president and CEO, Margaret Anderson, the FDA approved 35 new therapeutic in 2010; up from the 22 it approved a year earlier. She summed up this growth due to their being strong science, industry leadership and regulatory flexibility. However, Anderson summarized the current pipeline outlook saying, “There is a pipeline issue - they can only review and approve what comes before them. The reason for high approval rate was that these products were in the pipeline years ago. Today, the pipeline isn't as robust. The challenge may be in having too many opportunities but no way to choose or implement or prioritize them.”  An excited group of panelists, which included FDA Commissioner Margaret Hamburg, spent the next 90 minutes tackling this topic.

John Mendlien, who is at the helm of several small biotechnology companies, suggested that the focus needs to be on saving time - not necessary about money. To this point, Susan Desmond Hellmann, chancellor of the University of California at San Diego, added that the field needs biomarkers of disease and drug response desperately and the creation of animal models are areas where academics can lead the way. She also reflected that the cost of doing science has lessened as innovation has increased; using the example of the cost of sequencing a person’s genome, dropping to about $2,000 per person from the $95 million it was not too long ago. “It will soon cost less than an MRI, likely within the next year,” said Desmond-Hellmann. This comment led to a lively exchange between several of the panelists, including Mendlein who asked rhetorically and excitedly what the potential opportunities would exist for therapeutic development if every person in the US had their genome sequenced. Responding to this, at the behest of Anderson, Garry Neil, of Johnson & Johnson, interjected that while having data is great it needs to be collected and used in a meaningful way and the collection of such, if done by a private corporation, would provide them a competitive advantage.

Representing the patient advocacy perspective on the panel was Jeff Brewer, the president and CEO of the Juvenile Diabetes Research Foundation (JDRF). He offered these comments, “Incentives are needed for companies to invest in a specific area of research. Partnerships with companies are key for a non-profit foundation.” Brewer then outlined how JDRF has dozens of direct financial relationships and hundreds of other relationships with biotechnology and pharmaceutical companies. “You need to erode the competition between groups working on the same disease especially, when they fund the same researchers and work,” said Brewer. You can view the entire panel discussion online at FasterCures being clicking here or hitting play on the video above. 

Back to Basics: Patient Activism Thirty Years After HIV/AIDS

Another highlight of the first day was the “Back to Basics” panel organized by Susan Love, which can be viewed in its entirely be viewed at the FasterCures website. Warren Lammert, chairman and co-founder of the Epilepsy Therapy Project, and others were invited to provide advice to emerging non-profits on how best to organize and advocate. Lammert’s advice was that each organization needs to have clear ways to measure their success, otherwise you won’t have an impact. His organization for example has decided to focus on ensuring that the pipeline of potential treatments for epilepsy stays robust and therefore they fund projects that were unable to get funding elsewhere from government or other private sources.

ALS Therapy Development Institute, TDP-43 Mouse Model Partnership

Steve Perrin, president, CEO and CSO of the ALS Therapy Development Institute was also invited by the steering committee of this event to present an overview on the $1 million + collaboration between his organization and three others to charectorize an emerging model of neurodegeneration: the TDP43 mouse. The collaboration included both funding and scientific input from the Alzheimer's Drug Discovery Foundation, the Association for Frontotemporal Degeneration and the Muscular Dystrophy Association. Perrin suggested that such partnerships allow for the speeding of research and in turn eventually treatments by working across diseases at the same time rather than focusing each group to do the exact same experiment which would take longer and cost patients and donors more money.

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