ALS Therapy Development Institute (ALS TDI) CEO and Chief Scientific Officer Steve Perrin presented updates on the Institute’s drug development pipeline for ALS during the research symposium portion of a two-day ALS conference.
The MDA-supported nonprofit biotech’s 2010 Leadership Summit featured tours of the ALS TDI lab facilities in Cambridge, Mass., on Oct. 3. A research symposium, which was broadcast live over the Internet, followed on Oct. 4.
Read below for highlights of the conference or, to view the archived webcast, visit http://register.webcastgroup.com/l3/?wid=0761004105307.
ALS TDI — pipeline progress
Perrin reported that as ALS TDI searches for pharmaceutical partners to help move its lead therapeutic candidate, ALSTDI-00846, into clinical trials, work continues on other drugs of interest. These include:
ALSTDI-000876 (NTF5): Evidence suggests this drug is neuroprotective and promotes growth at the neuromuscular junction.
ALSTDI-000866 (apocynin) and ALSTDI-000896: Previous studies have produced intriguing results, but neither of these drugs conferred any benefit in testing at ALS TDI.
ALSTDI-000486 and ALSTDI-00903: Both drugs aim to modulate the immune system.
‘Hot topics’ in ALS
Fernando Vieira, ALS TDI director of in vivo validation, addressed several areas of ALS research, including:
Stem cells: Vieira commented on the growing popularity of induced pluripotent stem cells (iPS). He noted that a motor neuron line, created with ALS-affected cells that were regressed to the pluripotent stage and then prompted to develop into neurons, already has been produced for use in drug screening.
Immune system modulation: Although immune system involvement of neuron support cells called microglia has been implicated by some scientists for years, recent work at ALS TDI has demonstrated a role for other areas of the immune system as well.
Outside experts weigh in
Guest speakers at the symposium included Merit Cudkowicz, an MDA research grantee and director of the MDA/ALS Center at Massachusetts General Hospital in Boston; Gilmore O’Neill, vice president of experimental neurology at Biogen Idec, headquartered in Weston, Mass.; and Clive Svendsen, director of the Cedars-Sinai Regenerative Medicine Institute in Los Angeles.
Cudkowicz, who also is a member of MDA’s Medical Advisory Committee, said the pace of ALS research is increasing as the discovery of more ALS-associated genes brings in more scientists. She described the ins and outs of clinical trials, including trial design, phases, requirements, oversight, purposes and ALS-specific challenges.
Despite the various difficulties associated with testing candidate therapeutics, “Clinical trials are critical to finding the cure for ALS,” Cudkowicz said.
O’Neill reported on the need to identify and develop ALS clinical biological indicators, called “biomarkers,” as a means to deriving more reliable results in clinical trials.
The goal in developing therapeutics is to confirm that the drugs are being delivered to the central nervous system, that they are engaging the intended targets, and that they’re getting the desired biological and biochemical response, O’Neill said. Ultimately, biomarkers can answer this question for clinicians and researchers: Is what I think I’m seeing actually the reality of the situation?
Svendsen presented data on an area of study that has “exploded” over the last few years: stem cell therapy in ALS.
The first clinical trial to study injection of neural stem cells into the spinal cords of ALS patients, funded by Maryland biotherapeutics company Neuralstem, opened at the MDA/ALS Center at Emory University in Atlanta, in September 2009.
Svendsen noted that a second trial already is in the planning stages as a follow-up to the trial at Emory. Preclinical safety studies are expected to begin in the next few months. Patient enrollment is not yet open.