Can a re­for­mu­la­tion of an old drug on the mar­ket for asth­ma, al­ler­gy and mas­to­cy­to­sis pro­tect against liv­er dis­ease, Alzheimer’s, and amy­otroph­ic lat­er­al scle­ro­sis?

A team from Mass­a­chu­setts Gen­er­al Hos­pi­tal is mak­ing a case for the lat­ter two. Hav­ing re­cent­ly found an in­jec­tion of cro­molyn sodi­um ef­fec­tive in in­hibit­ing amy­loid be­ta (Aβ) ag­gre­ga­tion in vit­ro and in mouse mod­els, re­searchers set out to in­ves­ti­gate whether the same com­pound can achieve the same in ALS.

Their con­clu­sion, pub­lished on Na­ture’s open ac­cess jour­nal Sci­en­tif­ic Re­ports:

Our re­sults in­di­cate that cro­molyn sodi­um treat­ment sig­nif­i­cant­ly de­layed the on­set of neu­ro­log­i­cal symp­toms, and im­proved deficits in PaGE per­for­mance in both male and fe­male mice, how­ev­er, there was on­ly an ef­fect on sur­vival in fe­male mice.

While the pre­cise eti­ol­o­gy of ALS re­mains poor­ly un­der­stood, one the­o­ry pro­pos­es that neu­roin­flam­ma­to­ry process­es are im­pli­cat­ed in its ini­ti­a­tion and pro­gres­sion.

As cro­molyn in­hibits mast cell de­gran­u­la­tion, the sci­en­tists at Mass Gen­er­al hy­poth­e­sized that it could con­vert im­mune cells in the brain, in­clud­ing mi­croglia and as­tro­cytes, from a pro-in­flam­ma­to­ry to an an­ti-in­flam­ma­to­ry state as well as re­duc­ing the lev­els of cy­tokines and chemokines.

Af­ter com­par­ing the ef­fects of once-dai­ly in­jec­tions of cro­molyn ver­sus a place­bo in wild type mice and mice car­ry­ing a ge­net­ic mu­ta­tion for ALS, re­spec­tive­ly, the re­searchers came up emp­ty on the mi­croglia and as­tro­cytes the­o­ry. But they did find low­er lev­els of pro-in­flam­ma­to­ry cy­tokines/chemokines in the spinal cord and plas­ma — in ad­di­tion to ob­serv­ing that the trans­genic mice treat­ed with cro­molyn sodi­um had the high­est mo­tor neu­ron counts among the four groups.

“Our study sup­ports the no­tion that in­flam­ma­tion has a sig­nif­i­cant role in the pro­gres­sion of ALS and there­fore ex­plor­ing an­ti-in­flam­ma­to­ry treat­ments may be of great val­ue for de­vel­op­ing an ef­fec­tive treat­ment,” said Ghaz­a­leh Sadri-Vak­ili, lead au­thor of the study and di­rec­tor of the Neu­roEpi­ge­net­ics Lab­o­ra­to­ry at Mass Gen­er­al, in a state­ment.

Sadri-Vak­ili has pre­vi­ous­ly not­ed that the neu­rol­o­gy de­part­ment at Mass Gen­er­al has a plan in place to get the drug — pro­vid­ed by AZTher­a­pies — in­to the clin­ic for ALS.

Un­like the for­mu­la­tions of cro­molyn cur­rent­ly avail­able through pre­scrip­tion and over the counter, which are ab­sorbed through lung and nasal in­hala­tion or in­gest­ed drops, the ver­sion in­ject­ed in­to mice in the study can be “ful­ly avail­able in the blood­stream” and cere­brospinal flu­id, ac­cord­ing to the biotech.

AZTher­a­pies is con­duct­ing a Phase III tri­al of an­oth­er for­mu­la­tion of cro­molyn, com­bined with oral ibupro­fen, to treat ear­ly Alzheimer’s. It is un­clear whether any clin­i­cal pro­grams are in place to test cro­molyn sodi­um in pri­ma­ry scle­ros­ing cholan­gi­tis af­ter Bay­lor Scott and White Health re­searchers re­port­ed it de­creased bil­iary pro­lif­er­a­tion and fi­bro­sis in mice.

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.

Boehringer Ingelheim reported human pharma sales of €18.5 billion on Wednesday, led by type 2 diabetes and heart failure drug Jardiance and pulmonary fibrosis med Ofev. Jardiance sales reached €5.8 billion, growing 39% year over year, while Ofev took in €3.2 billion, notching its own 20.6% annual jump.

However, Boehringer is also looking ahead with its pipeline, estimating “In the next seven years the company expects about 20 regulatory approvals in human pharma.”

The FDA today approved Emergent BioSolutions’ Narcan brand naloxone nasal spray for over-the-counter sales. The nod was expected and comes on the heels of a unanimous 19-0 advisory committee vote in favor of approval last month.

The move to OTC means the opioid overdose reversal agent will now be available on grocery, convenience and gas stations shelves, as well as potentially for purchase online.

The discovery arm of the healthcare investment player Deerfield Management Company is getting a major financial boost.

Deerfield Discovery and Development will receive $25 million, over five years, from the state of New York and Empire State Development, a state organization that offers grants, loans and other assistance to companies. Another $25 million will come from Deerfield Management.

The funds will go toward the buildout of a 6,000-square-foot lab space at Cure, a healthcare campus founded by Deerfield at 345 Park Ave. South in New York City. The money will also be used for purchasing equipment and software, operating costs and other items.